VIRAL VECTOR & GENE EDITING CORE
Whether you are an experienced researcher or just getting started with gene delivery and genome engineering, the Florida Institute for Pediatric Rare Diseases (IPRD) Viral Vector and Gene Editing Core (VVGEC) service is here to help. We offer expert support, custom services, and a friendly, collaborative approach to meet your scientific goals.
The IPRD Viral Vector and Gene Editing Core is a full-service facility specializing in the design, development, and production of viral vectors and gene editing tools. We support research projects of all types and sizes, and our services are available to academic, clinical, and industry partners.
Our team brings extensive expertise in:
We offer everything from small pilot batches to large-scale vector production, along with consultation and project design support every step of the way.
Core Services Overview
Here are some of the ways we can support your research:
Viral Vector Services
- Design, construction, and cloning of plasmids for gene delivery
- Ready-to-use vector backbones for your gene of interest
- Production of lentivirus, AAV, retrovirus, and adenovirus vectors
- Support for in vitro and in vivo applications
- Flexible production scales: small, mid, or large batches
- Naïve and reporter constructs in both AAV and lentiviral formats
- Robust quality control, including plasmid sequencing and safety testing.
Gene Editing Services
- Custom CRISPR/Cas9 design and validation
- Creation of knockout, knock-in, and conditional models (KO, KI, CKO)
- Generation of genetically modified stable cell lines
- Access to a large library of RNA interference (RNAi) vectors and CRISPR tools for conventional, base, prime, and epigenome editing
Optogenetics
- Access to more than 300 optogenetic vectors from leaders in the field ( Karl Deisseroth , Bryan Roth , Ed Boyden , and others)
- Tools to control cell activity with light in complex tissues
- Consultation on vector selection and application design
Protein and Antibody Engineering
- Custom design of antibodies, enzymes, and protein switches
- Engineering of proteins with novel binding or signaling capabilities
- Support from early-stage discovery through stable cell line development
Personalized Project Support
Our team works closely with investigators to design and deliver custom solutions. We provide one-on-one consultations to:
- Recommend appropriate viral vector platforms and gene editing tools
- Select optimal serotypes or pseudotypes for your target tissues or cells
- Help integrate CRISPR tools with transgenic animal models
- Support every step from concept to implementation
Why Choose Us
Get Started
Whether you need a single batch of virus or full-service support for a complex genome editing project, we are here to help.
Contact us today to schedule a consultation, request a quote, or learn more about how our services can accelerate your research.
The IPRD Viral Vector and Gene Editing Core (VVGEC) facilitates the use of virus-mediated tools for gene transfer by investigators across diverse fields of study, such as systems neuroscience, stem cell biology, metabolism, aging, cancer biology and others.
VVGEC provides services to investigators in the construction, purification, and titering of various viral vectors, including lentiviral vectors (LVs) and adeno-associated vectors (AAVs) for research use.
Services Overview
Viral Vector Construction
Generating Viral Particles
Consulting
Adeno-Associated Vectors
About Adeno-Associated Vector (AAV) – Why it’s the platform-of-choice for preclinical and gene therapy research
Adeno-associated vector (AAV) is a ssDNA, non-pathogenic, non-harmful viral delivery system that has emerged as a superb research tool and leading platform for genetic cargo delivery in preclinical and clinical applications. Several distinct attributes make AAV the gold-standard vector for both academic and industry investigators:
- Non-Pathogenic, Non-Harmful; High Safety: AAV is not currently known to cause any disease in humans, making it a preferred choice for in vitro and in vivo delivery.
- Low Immunogenicity and Toxicity: AAV vector transduction results in a minimal immune response, supporting higher dosing, stable and long-term gene expression, and minimal off-target side effects for research-grade and clinical-grade indications.
- Broad and Modifiable Tropism: AAV can efficiently infect both dividing and non-dividing cells—which greatly fit into a broad range of applications.
- Stable, Durable and Long Term Expression: AAV vectors can drive long-term, robust and stable gene expression without integrating the transgene-of-interest into the host chromosome, such reducing safety concerns and enabling effective therapeutic outcome.
- Serotype-Driven Specificity: AAV can efficiently infect wide range of cells and tissues, and its capsids can be easily modified via serotype selection, such offering precise tissue or cell-type targeting for research use and gene therapy.
Adeno-Associated Vector Prep Summary
| |
|
|
|
|
Production Time | |
|
| Small AAV preps (I n Vitro Grade) |
In Vitro |
≥ 5 × 10¹¹ vg/mL | 10 mL |
1 mL |
7 days |
$360 | $450 |
| Large AAV preps (I n Vitro Grade) |
In Vitro |
≥ 5 × 10¹¹ vg/mL | 100 mL |
10 µL |
7 days |
$900 | $1125 |
| Small AAV Preps (In Vivo Grade) |
In Vivo |
≥ 5 × 10¹² vg/mL | 200 µL |
25 µL |
14 days |
$660 | $825 |
| Mid (Standard) AAV Preps (In Vivo Grade) |
In Vivo |
≥ 5 × 10¹² vg/mL | 50 µL |
25 µL |
14 days |
$2040 | $2550 |
| Large AAV Preps (In Vivo Grade) |
In Vivo |
≥ 1 × 10¹² vg/mL | 200 µL |
25 µL |
21 days |
$5976 | $7440 |
| Mega AAV Preps (In Vivo Grade) |
In Vivo |
≥ 1 × 10¹² vg/mL | 1 mL |
25 µL |
28 days |
|
$15250 |
Internal FSU user rates are available, please contact us directly for a quote.
Find the best way to get help and connect with FSU’s Viral Vector Gene Editing Core. Fill out a short form to place an order with us or simply send an email with your request to viral.core@med.fsu.edu
Order Form
Retroviral Vectors
What are Retroviral Vectors?
Retroviral vectors are derived from retroviruses, a family of RNA viruses that replicate through a DNA intermediate using reverse transcription. The viral RNA is converted into DNA by the enzyme reverse transcriptase, and this DNA is then integrated into the host genome by integrase.
Applications in Gene Therapy
Retroviral vectors have a wide range of applications, including:
- Gene Therapy: Retroviruses are commonly employed in gene therapy applications to deliver therapeutic cargo to the desired cell population
- Cancer Treatment: Retroviruses are employed in advanced therapies like CAR T-cell therapy, where T-cells are modified to find and destroy cancer cells.
- Vaccine Development:They are also being explored for use in vaccine development, for diseases where traditional vaccines are ineffective.
- Preclinical Research:Retroviruses are commonly used in basic research to introduce gene products into in vitro systems or animal models, facilitating studies on gene function and regulation.
Advantages of Retroviral Vectors
- Ability to Infect Dividing Cells:retroviral vectors are capable of transducing dividing cells with high efficiency; they are unable to transduce non-dividing or slow- pace dividing cells, which can be an advantage for some applications; e.g. labeling and tracing of pre-mitotic neurons in the CNS
- Stable Gene Expression:Once integrated into the host genome, retroviral vectors can provide stable and long-term expression of the therapeutic gene.
- Low Immunogenicity:Engineered, recombinant retroviral vectors typically exhibit low immunogenicity, reducing the likelihood of an adverse immune response.
Safety Considerations
While retroviral vectors offer significant therapeutic potential, there are important safety concerns, including:
- Immune Reactions: Although designed to be less immunogenic, there is still a risk of the host immune system recognizing and reacting to the viral components.
- Undesired Integration:Retroviral vectors are integrating systems. The undesired integration may increase a risk of insertional mutagenesis, oncogenicity and other related toxicities.
Conclusion: Retroviral vectors represent a promising avenue in gene therapy and molecular biology, with ongoing research aimed at optimizing their safety and efficacy. Their unique ability to deliver genetic material into a wide variety of cell types makes them invaluable tools in both research and clinical applications.
Retroviral Vector Production
The IPRD Viral Vector and Gene Editing Core offers high-quality retroviral vectors suitable for a variety of research applications. Our retroviral vectors are designed for stable integration into dividing cells and are produced under rigorous quality control procedures.
Titering and Aliquoting
All retroviral preparations are titered using:
- Flow cytometry or colony-forming assays when reporter or selection markers are present
- Real-time PCR (qPCR) on transduced cells when such markers are absent
Aliquots are provided in 1 mL, 10 mL, or 25 µL volumes depending on prep type. All vectors undergo rigorous quality control to ensure consistency and safety.
Retroviral Vector Prep Summary
| Prep Type |
Grade |
Titer |
Total Volume |
Aliquot Size |
Production Time | Academic Cost |
Non-Academic Cost |
| Small Non-Concentrated |
In Vitro |
≥ 1 × 10⁷ vg/mL |
10 mL |
1 mL |
7 days |
$360 | $450 |
| Large Non-Concentrated |
In Vitro |
≥ 1 × 10⁷ vg/mL |
100 mL |
10 µL |
7 days |
$900 | $1125 |
| Concentrated (In Vitro Grade) |
In Vitro |
≥ 2.5 × 10⁹ vg/mL |
200 µL |
25 µL |
14 days |
$1080 | $1350 |
| Small Concentrated (In Vivo Grade) |
In Vivo |
≥ 2.5 × 10⁹ vg/mL |
50 µL |
25 µL |
14 days |
$895 | $1112 |
| Medium Concentrated (Standard In Vivo) |
In Vivo |
≥ 2.5 × 10⁹ vg/mL |
200 µL |
25 µL |
21 days |
$1800 | $2250 |
| Large Concentrated (In Vivo Grade) |
In Vivo |
≥ 5 × 10⁹ vg/mL |
1 mL |
25 µL |
28 days |
|
$9000 |
Internal FSU user rates are available, please contact us directly for a quote.
Find the best way to get help and connect with FSU’s Viral Vector Gene Editing Core. Fill out a short form to place an order with us or simply send an email with your request to viral.core@med.fsu.edu
Order Form
Lentiviral Vectors
What are Lentiviral Vectors?
Lentiviral vectors are powerful tools in basic research and gene therapy, derived from lentiviruses, a subgroup of retroviruses.
Applications in Gene Therapy
Lentiviral vectors have a wide range of applications, including:
- Gene Therapy: They are used to treat genetic disorders by delivering therapeutic cargo to correct defective genes in patients.
- Cancer Treatment: Lentiviral vectors are employed in advanced therapies like CAR Tcell therapy, where T-cells are modified to find and destroy cancer cells.
- Vaccine Development:They are also being explored for use in vaccine development, for diseases where traditional vaccines are ineffective.
- Preclinical Research:Lentiviral vectors are commonly used in basic research to introduce gene products into in vitro systems or animal models, facilitating studies on gene function and regulation.
Advantages of Lentiviral Vectors
- Ability to Infect Non-Dividing Cells:Unlike many other viral vectors, lentiviral vectors can infect both dividing and non-dividing cells, broadening their application
- Stable Gene Expression:Once integrated into the host genome, lentiviral vectors can provide stable and long-term expression of the therapeutic gene.
- Low Immunogenicity:Engineered lentiviral vectors typically exhibit low immunogenicity, reducing the likelihood of an adverse immune response.
Safety Considerations
While lentiviral vectors offer significant therapeutic potential, there are important safety concerns, including:
- Insertional Mutagenesis:The integration of the viral DNA into the host genome can disrupt essential genes or activate oncogenes, potentially leading to cancer.
- Immune Reactions: Although designed to be less immunogenic, there is still a risk of the host immune system recognizing and reacting to the viral components.
Conclusion: Lentiviruses represent a promising avenue in gene therapy and molecular biology, with ongoing research aimed at optimizing their safety and efficacy. Their unique ability to deliver genetic material into a wide variety of cell types makes them invaluable tools in both research and clinical applications.
The recommendation of National Institute of Health (NIH) to test concentrated-lentiviral vectors on the subject of appearance of replication-competent retroviruses (RCR) is attached in the document below. The service provided by VVGEC and is mandatory is highlighted here.
The IPRD Viral Vector and Gene Editing Core offers high-quality lentiviral vectors in multiple grades to support a range of in vitro and in vivo applications. All vector preps are quality-controlled and can be customized upon request.
Titering and Aliquoting
All viral preps are rigorously titered for accuracy and reliability:
- Flow cytometry or antibiotic-resistant colony counts are used for vectors expressing appropriate markers.
- For marker-free vectors, real-time PCR (qPCR) on transduced cells is used.
- Vectors are aliquoted in 1 mL, 10 mL, or 25 µL volumes depending on prep type.
Lentiviral Vector Prep Summary
| Prep Type |
Grade |
Titer |
Total Volume |
Aliquot Size |
Production Time | Academic Cost |
Non-Academic Cost |
| Small Non-Concentrated |
In Vitro |
≥ 2.5 × 10⁷ vg/mL |
10 mL |
1 mL |
7 days |
$360 |
$450 |
| Large Non-Concentrated |
In Vitro |
≥ 2.5 × 10⁷ vg/mL |
100 mL |
10 µL |
7 days |
$900 |
$1125 |
| Concentrated (In Vitro Grade) |
In Vitro |
≥ 5 × 10⁹ vg/mL |
200 µL |
25 µL |
14 days |
$1080 |
$1350 |
| Small Concentrated (In Vivo Grade) |
In Vivo |
≥ 5 × 10⁹ vg/mL |
50 µL |
25 µL |
14 days |
$1080 |
$1350 |
| Medium Concentrated (Standard In Vivo) |
In Vivo |
≥ 5 × 10⁹ vg/mL |
200 µL |
25 µL |
21 days |
$1792 |
$2231 |
| Large Concentrated (In Vivo Grade) |
In Vivo |
≥ 5 × 10⁹ vg/mL |
1 mL |
25 µL |
28 days |
$1080 |
$1350 |
Find the best way to get help and connect with FSU’s Viral Vector Gene Editing Core. Fill out a short form to place an order with us or simply send an email with your request to viral.core@med.fsu.edu
Order Form
CRISPR/Cas9 Gene-editing Tools
CRISPR ( C lustered R egularly I nterspaced S hort P alindromic R epeats) is a powerful tool that enables scientists to manipulate the genome with ease. Over the past decade, scientists have expanded the CRISPR/Cas arsenal to include many different types of genetic manipulations, including gene knock-outs, knock-ins, point mutations, base changes, and small and large insertions and deletions. For more information on the wide range of CRISPR technologies, please email us viral.core@med.fsu.edu
The advanced CRISPR/Cas tools will support your research as follows;
- Loss-of-function studies aimed to generate complete and permanent loss of gene expression or function (knock-out)
- Introduce a fragment-of-interest into a specific genomic locus (knock-in)
- Create a specific mutant allele of a gene (point mutant)
- Increase or decrease expression of a target gene (epigenome editing)
Lentiviral Vectors - backbones
- pBK109- short version derived from pLentiCRISPR/v2 ready-for-gRNA-cloning (BsmBI)
- pBK301- short version of pLentiCRISPR/v2 with 2 Sp1 binding sites upstream of U6p- gRNA cloning site BsmBI
- pBK175- short version of pLentiCRISPR/v2 with 4 Sp1 binding sites upstream of U6p- gRNA cloning site BsmBI
- pBK110- pLentiCRISPR/v2 Luciferase-gRNA
- pBK104- pLentiCRISPR/v2 GABA a2-subunit-gRNA
- pBK83- pLentiCRISPR/v2 HDAC1-1 –gRNA
- pBK84-pLentiCRISPR/v2 HDAC1-2 –gRNA
- pBK85-pLentiCRISPR/v2 HDAC1-3 – gRNA
- pBK86-pLentiCRISPR/v2 GFP1 – gRNA
- pBK87-pLentiCRISPR/v2 GFP2 – gRNA
- pBK88-pLentiCRISPR/v2 GFP3 – gRNA
- pBK189- pLentiCRISPR/v2 with GFP-gRNA
- pBK198- short version of pLentiCRISPR/v2 with 2 Sp1 binding sites with GFP-gRNA
- pBK180- rtTA3 Blasticidin third generation rtTA3 can be used for tet-Cas9 system (next plasmid)
- pBK185- tet- inducible Cas9- all-in-one short- with BsmBI for cloning sgRNA
- pBK195- tet- inducible Cas9- all-in-one short- with GFP-gRNA
- pCW-Cas9 Tet ON Plasmid #50661; Addgene- https://www.addgene.org/50661/
- pBK109easy -GFP pLenti-SpCas9gRNA with GFP all-in-one
- pBK97- SpCas9 nickase- all-in-one lentiviral backbone; titer-optimized backbone; ready for gRNA cloning BsmBI
- pBK456- dCas9- all-in-one lentiviral backbone; titer-optimized backbone; ready for gRNA cloning BsmBI
- pBK109easy -GFP pLenti-SpCas9gRNA with GFP all-in-one
- pBK109BL- short version of pLentiCRISPR/v2 gRNA cloning site BsmBI with blasticidin
- pBK114- pLenti-Cas9-GFP
- pLentiCRISPR/v2 Plasmid #52961; Addgene- https://www.addgene.org/52961/
- pLenti-multi-CRISPR; Plasmid #85402; Addgene- https://www.addgene.org/85402/
- LentiCRISPRv2-mCherry; Plasmid # 99154; Addgene- http://www.addgene.org/99154/
- pLenti-pU6-sgRNA Ef1alpha-Puro-T2A-BFP Plasmid #84832; Addgene- http://www.addgene.org/84832/
- pLenti-sgRNA; Plasmid #71409; Addgene- http://www.addgene.org/71409/
- pLenti-CRISPR.EFS.tRFP; Plasmid #57819; Addgene- http://www.addgene.org/57819/
- lentiCas9-Venus; Plasmid # 70267; Addgene- http://www.addgene.org/70267/
- pL-CRISPR.EFS.GFP Plasmid #57818; Addgene- https://www.addgene.org/57818/
- lentiCas9-Blast Plasmid #52962; Addgene- https://www.addgene.org/52962/
- lentiCas9-Puro Plasmid #52963; Addgene-http://www.addgene.org/52963/
- lentiCas9-EGFP; Plasmid #63592; Addgene- https://www.addgene.org/63592/
- lentiCas9n(D10A)-Blast; Plasmid #63593; Addgene- http://www.addgene.org/63593
- lenti-dCAS-VP64-Blast; Plasmid #61425; Addgene- http://www.addgene.org/61425/
- pHAGE TRE dCas9-VP64; Plasmid #50916; Addgene- http://www.addgene.org/50916/
- lenti-TRE-KRAB-dCas9-IRES-BFP; Plasmid #85449; Addgene- http://www.addgene.org/85449/
- plenti- hUbC-dCas9 VP64-T2A-GFP; Plasmid #53192; Addgene- http://www.addgene.org/53192/
- pKLV-U6gRNA(BbsI)-PGKpuro2ABFP; Plasmid #50946; Addgene- http://www.addgene.org/50946/
- plentiSAMv2; Plasmid #75112; Addgene- http://www.addgene.org/75112/
- pHR-SFFV-KRAB-dCas9-P2A-mCherry; Plasmid #60954; Addgene- http://www.addgene.org/60954/
- pLV-dCas9-KRAB-PGK-Hyg- Plasmid #83890; Addgene- http://www.addgene.org/83890/
- pLV hU6-sgRNA hUbC-dCas9-KRAB-T2a-GFP; Plasmid #71237; Addgene- http://www.addgene.org/71237/
- pHAGE EF1α dCas9-KRAB; Plasmid #50919; Addgene- http://www.addgene.org/50919/
- lenti sgRNA-MS2-Zeo; Plasmid #61427; Addgene- http://www.addgene.org/61427/
- lenti sgRNAMS2- puro optimized backbone; Plasmid #73797; Addgene- http://www.addgene.org/73797/
- pHAGE-TO-nmdCas9-3XGFP; Plasmid #64109; Addgene- http://www.addgene.org/64109/
- pLV-dCas9-p300-P2A-Puro; Plasmid #83889; Addgene- http://www.addgene.org/83889/
- Lenti_sgRNA-EFS-GFP; Plasmid #65656; Addgene- http://www.addgene.org/65656/
- Lenti-AsCpf1-Blast; Plasmid #84750; Addgene- http://www.addgene.org/84750/
- LentiCRISPRv2Cre; Plasmid #82415; Addgene- http://www.addgene.org/82415/
- Exp_v-pcDNA3.1-hAsCpf1; Plasmid #69982; Addgene- https://www.addgene.org/69982/
- Exp_v-pSimpleII-U6- gRNA cloning site BsmBI-NLS-NmCas9-HA-NLS plasmid # 47868; Addgene https://www.addgene.org/47868/
- Exp_v-pSaCas9_GFP; Plasmid # 64709; Addgene- https://www.addgene.org/64709/
- Exp_v-Csy4-T2A-Cas9-NLS; Plasmid # 53371; Addgene- https://www.addgene.org/53371/
- Exp_v-ppcDNA3.1-hFnCpf1; Plasmid #69976; Addgene- https://www.addgene.org/69976/
- Exp_v-eSpCas9(1.1)- Plasmid #71814; Addgene-https://www.addgene.org/71814/
- Exp_v-SpCas9-HF1 (high fidelity Cas9); Plasmid #72247; Addgene- https://www.addgene.org/72247/
AAVs
- AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA Plasmid # 61591; Addgene- https://www.addgene.org/61591/
- BK- pAAV-EFS-NC- SpCas9-NLS-Poly(A)
- BK- pAAV-CMV-SpCas9- NLS-Poly(A)
- BK- pAAV-hSyn -SpCas9- NLS-Poly(A)
- BK- pAAV- -SpCas9- NLS-Poly(A)
- pAAV- nEF promoter- Cas9; Plasmid #87115; Addgene- http://www.addgene.org/87115/
- pAAV-pMecp2-SpCas9 Plasmid #60957 http://www.addgene.org/60957/
- pX602-AAV-TBG::NLS-SaCas9-NLS-HA-OLLAS-bGHpA;U6::BsaI-sgRNA Plasmid #61593; Addgene- http://www.addgene.org/61593/
- pX601-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA; Plasmid #61591; Addgene- http://www.addgene.org/61591/
- AAV-NFS-NC: NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA
- AAV-Syn: NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA
- pX600-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA; Plasmid #61592; Addgene- http://www.addgene.org/61592/
- pJEP12-AAV-H1/TO(dox-regulated)-L-sgRNA(Empty)-CMV-TetR-P2A-eGFP-KASH-pA; Plasmid #82704; Addgene- http://www.addgene.org/82704/
- pX603-AAV-CMV::NLS-dSaCas9(D10A,N580A)-NLS-3xHA-bGHpA; Plasmid #61594; Addgene- http://www.addgene.org/61594/
- AAV-NFS-NC:NLS-dSaCas9(D10A,N580A)-NLS-3xHA-bGHpA
- AAV-Syn:NLS-dSaCas9(D10A,N580A)-NLS-3xHA-bGHpA
- AAV-MeCp2:NLS-dSaCas9(D10A,N580A)-NLS-3xHA-bGHpA
- AAV-NFS-NC:NLS-dSaCas9(D10A,N580A)-NLS-3xHA-bGHpA
- AAV-NFS-NC:NLS-dSaCas9Nickase-NLS-3xHA-bGHpA
- AAV-CMV:NLS-dSaCas9Nickase-NLS-3xHA-bGHpA
- AAV-CAG:NLS-dSaCas9Nickase NLS-3xHA-bGHpA
- AAV:ITR-U6-sgRNA(backbone)-hSyn-Cre-2A-EGFP-KASH-WPRE-shortPA-ITR; Plasmid #60231; Addgene- http://www.addgene.org/60231/
- AAV:ITR-U6-sgRNA(backbone)-pEFS-Rluc-2A-Cre-WPRE-hGHpA-ITR; Plasmid #60226; Addgene- http://www.addgene.org/60226/
- AAV:ITR-U6-sgRNA(Kras)-U6-sgRNA(p53)-U6-sgRNA(Lkb1)-pEFS-Rluc-2A-Cre-shortPA-KrasG12D_HDRdonor-ITR (AAV-KPL); Plasmid #60224; http://www.addgene.org/60224/
- BK- pAAV-TRE – SpCas9-NLS-Poly(A)
- BK- pAAV-TRE- SaCas9-NLS-Poly(A)
- BK- pAAV-TRE – dSpCas9-NLS-Poly(A)
- BK- pAAV-TRE – dSaCas9-NLS-Poly(A)
- BK- pAAV-TRE – SpCas9Nickase-NLS-Poly(A)
- BK- pAAV-TRE – SaCas9Nickase-NLS-Poly(A)
The IPRD Viral Vector and Gene Editing Cores deliver advanced solutions for biomedical research and therapeutic development. Our expertise in CRISPR/Cas technologies, viral vector engineering, and customized cell and animal model generation empowers investigators to accelerate discovery and translational applications.
We offer a comprehensive portfolio of services that includes the design and production of CRISPR/Cas vectors, generation of genetically engineered cell lines, and customized editing strategies using the latest technologies such as base editing, prime editing, and epigenome editing. Our team is here to partner with you—whether you are developing basic research tools, building disease models, or pursuing therapeutic targets.
- Gene Knockout Cell Line Services
Our core provides fully customizable CRISPR/Cas9-based knockout services in a wide range of mammalian cell lines, including difficult-to-transfect and tumor-derived lines. We deliver functionally validated, long-term stable KO models to researchers at Florida State University and to institutions and companies worldwide.
Our platform includes:
- High-throughput gRNA screening and optimized RNP delivery
- Single- and multi-gene knockout strategies
- Targeted fragment deletions
- End-to-end support from gRNA design to functional validation
Standard workflow:
- Host Cell Characterization
Includes clonability assays, antibiotic resistance profiling, and optimization of transfection/transduction. - gRNA Design & KO Vector Construction
Guides are designed to target conserved exons or critical functional domains of the gene-of-interest. - Transfection & Transduction
Using optimized techniques and house-developed lentiviral and adeno-associated vectors. - Clone Screening
Selection via antibiotic resistance or FACS; expansion of monoclonal or polyclonal populations. - Validation
Characterization via Western blotting, qPCR, ELISA, or reporter assays.
- Specialized Gene Editing Services
Multiplexed KnockoutsGenerate cell lines with multiple gene deletions using pooled or sequential editing strategies.
Disease ModelingCreate precise genetic alterations to model rare or common human diseases in vitro.
Custom CRISPR LibrariesDesign and produce small- to large-scale gRNA libraries for functional genomics studies.
Nuclease Activity ValidationQuantify and optimize CRISPR nuclease performance using a range of molecular and cellular assays.
- Base Editing and Prime Editing
We offer advanced genome editing platforms based on base editors and prime editors—technologies that allow precise, single-nucleotide changes without introducing double-stranded breaks.
Base Editing
Our cytidine and adenine base editors enable targeted C-to-T or A-to-G conversions with minimal off-target effects. These editors are delivered using viral vectors customized for your cell type and application.
- Editing window: 6–10 nucleotides
- Delivery via AAV, lentivirus, or electroporation
- Applications: point mutation correction, functional SNP studies, regulatory region targeting
Prime Editing
We design and deliver pegRNA constructs that enable precise insertions, deletions, or substitutions.
- pegRNA components: spacer, scaffold, reverse transcription template (RTT), primer binding site (PBS)
- nCas9-reverse transcriptase system enables templated DNA repair
- Suitable for applications requiring programmable, high-fidelity edits
With over 15 years of experience in guide RNA design, viral delivery, and construct engineering, we support projects across a wide range of biological systems.
- Epigenome Editing
Epigenetic modifications enable control of gene expression without altering the underlying DNA sequence. We offer full-service epigenome editing platforms using dCas9-based tools fused to:
- DNA methyltransferases and demethylases
- Histone acetyltransferases and deacetylases
Workflow includes:
- Target Identification – Defining loci relevant to gene regulation or disease
- Tool Construction – Building custom dCas9 fusion proteins
- Vector Development – Cloning into expression vectors for delivery
- Delivery – Viral, electroporation, or nanoparticle-based methods
- Selection & Expansion – Isolation of modified clones
- Validation – ChIP, bisulfite sequencing, and gene expression profiling
- Vector and Construct Resources
We maintain an extensive collection of >3,000 in-house optimized CRISPR/Cas constructs for gene knockout, base editing, and more. These are:
- Available directly from the core
- Deposited in the Addgene repository ( https://www.addgene.org )
- Linked to the NGVB repository ( https://ngvbcc.org/ReagentRepository )
Our design and production workflows are supported by 10 patent applications and numerous peer-reviewed publications and funded grants.
Let’s CollaborateWe welcome collaborations with academic, government, and industry partners. Whether your goal is to explore gene function, build disease models, or develop precision therapeutics, our core is ready to support your success.
Contact us today to discuss your project needs and receive a customized consultation.
Find the best way to get help and connect with FSU’s Viral Vector Gene Editing Core, simply send an email with your request to viral.core@med.fsu.edu
IPRD Viral Vector and Gene Editing Core owns an extensive collection of optogenetic and gene editing constructs.
Please note – we request that your publication acknowledge the donor of the reagent and the IPRD Viral Vector and Gene Editing facilities.
IPRD Viral Vector and Gene Editing Core has the following collection of unique and valuable tools:
Boris Kantor, Ph.D., https://www.addgene.org/Boris_Kantor/ ; Linked to the NGVB repository (https://ngvbcc.org/ReagentRepository)
Karl Deisseroth, M.D., Addgene: Karl Deisseroth Lab Materials ; https://www.addgene.org/Karl_Deisseroth/
Ed Boyden, Ph.D., Addgene: Edward Boyden Lab Materials ; https://www.addgene.org/Edward_Boyden/
Brian Roth, M.D., Addgene: Bryan Roth Lab Materials
; https://www.addgene.org/Bryan_Roth/
CRISPR/Cas tools Addgene: CRISPR Plasmids and Resources ; https://www.addgene.org/crispr/ ;
Standard Operating Procedures
(Rev. 6/2025)
Biosafety Plan
(Rev. 6/2025)
CLONING SERVICE
The IPRD Viral Vector and Gene Editing Core is pleased to offer an expanded and cost effective Molecular Cloning services. We would be happy to help with custom gene cloning and vector construction, shRNA, CRISPR/Cas9 synthesis, plasmid DNA and RNA purification, and other projects.
We have proven expertise in designing and producing complex DNA constructs for nearly any purpose.
Custom Gene Cloning and Vector Construction
- Cloning of cDNA, ORFs from any species in the lentiviral and AAV vector backbones.
- Protein-expression vectors for bacterial, and mammalian cell expression.
- We supply sequence-confirmed plasmid DNA and plasmid maps.
- Cloning using restriction enzyme digestion: sticky-end, blunt-end cloning.
- Cloning using PCR amplification: TA cloning or blunt-end cloning.
- Cloning by Gene Synthesis: we work with The GeneArt™ Gene Synthesis service.
http://www.thermofisher.com/us/en/home/life-science/cloning/gene-synthesis/geneart-gene-synthesis.html to synthesize an DNA fragments and oligos.
- Cloning by Gibson Assembly: to assemble several DNA fragments in correct order for gene synthesis, synthetic biology, and novel expression vectors.
- Cloning by RT-PCR: Isolated RNA/mRNA from any tissue is reverse transcribed and cloned into a plasmid-of-interest.
- Viral Expression Vectors: the core has an extensive collection of in-house AAV, Lentiviral and Retroviral vector backbones, with combination of heterologous envelopes, genes, promoters and tags.
- Gene Targeting Vectors: Vectors for homologous recombination, knock-in, knock-out, Cre, FLP, and Flox constructs for genome integration.
- Inducible Expression Vectors: Conditional and inducible systems for a temporal and spatial controlled activation of genes including Tet-On, Tet-Off, and tamoxifen-inducible.
- Cloning of miRNA, mi-shRNA and shRNA into a plasmid-of-interest- pol II or pol III-promoter systems are available for transient and stable expression.
Mutagenesis: Additions, deletions, substitutions, site-specific and random mutagenesis.
We can help you with your cloning strategy, whether we start from your ready-to-use insert or whether we help you to customize your vector construction.
Quality control: double-strand sequencing of full insert. The end-point plasmids released to investigator/used for vector production after we verify their sequence by digestion with restriction enzyme and Sanger sequencing. The integrity of inverted-terminal repeats (ITRs) of AAV-plasmids is routinely verified by digestion with restriction enzymes prior to vector production.
Check out our publicationsfocused on the development and optimization of viral vector tools paired with CRISPR/Cas9 systems for treatment of neurodegenerative diseases and disorders.
Publications
- Kantor B, Duke L, Bhide PG. CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges. Mol Ther Nucleic Acids. 2026 Mar 12;37(1):102786. doi: 10.1016/j.omtn.2025.102786. eCollection 2026 Mar 12. Review. PubMed PMID: 41496894; PubMed Central PMCID: PMC12767858.
- Kantor B, O’Donovan B, Chiba-Falek O. Trends and challenges of AAV-delivered gene editing therapeutics for CNS disorders: Implications for neurodegenerative disease. Mol Ther Nucleic Acids. 2025 Sep 9;36(3):102635. doi: 10.1016/j.omtn.2025.102635. eCollection 2025 Sep 9. Review. PubMed PMID: 40799507; PubMed Central PMCID: PMC12341529
- Kantor B, Odonovan B, Rittiner J, Hodgson D, Lindner N ,Guerrero S, Dong W, Zhang A, Chiba-Falek O. All-in-one AAV-delivered epigenome-editing platform: proof-of-concept and therapeutic implications for neurodegenerative disorders. Nature Communications 2024 Sept 23;15(1):7259. doi: 10.1038/s41467-024-50515-6.
- Kantor B, Odonovan B, Rittiner J, Lindner N, Dong W, Zhang A, Chiba-Falek O. All-in-one AAV-delivered epigenome-editing platform: proof-of-concept and therapeutic implications for neurodegenerative disorders bioRxiv [Preprint]. 2024 May 19:2023.04.14.536951. doi: 10.1101/2023.04.14.536951.
- Sun Z, Kantor B and Chiba-Falek O. Neuronal-type-specific epigenome editing to decrease SNCA expression: Implications for precision medicine in synucleinopathies Mol Ther Nucleic Acids 2023 Nov 24;35(1):102084.
- Rittiner J, Cumaran M, Malhotra S, and Kantor B. Therapeutic modulation of gene expression in the disease state: Treatment strategies and approaches for the development of next-generation of the epigenetic drugs Bioeng. Biotechnol., 2022 Sec. Preclinical Cell and Gene Therapy https://doi.org/10.3389/fbioe.2022.1035543
- Kantor B and Chiba-Falek O. Lentiviral vectors as the delivery vehicles for transduction into iPSCs: shortcomings and benefits 2021. Advances in Stem Cell Biology. Book Chapter (Methods in iPSC Technology Volume 9 in Advances in Stem Cell Biology 2021, Pages 79-100)
- Kantor B and Chiba-Falek O. Adeno-associated vectors for transduction into iPSCs: progress and perspectives 2021. Advances in Stem Cell Biology. Book Chapter (Methods in iPSC Technology Volume 9 in Advances in Stem Cell Biology 2021, Pages 68-78).
- Dong W and Kantor B. Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives. Viruses 2021, 13, 1288. https://doi.org/10.3390/v13071288
- Hunanyan AS, Kantor B, Puranam R, Elliott C, McCall A, Pagadala P, Wallace K, Poe J, Asokan A, Koeberl DD, ElMallah MK, Mikati M. AAV Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood (2021) Human Gene Therapy Apr;32(7-8):405-419. doi: 10.1089/hum.2020.191. Epub 2021 Feb 12
- MacDougall G, Brown LY, Kantor B, Chiba-Falek O. The path to progress preclinical studies of age-related neurodegenerative diseases: a perspective on rodent and hiPSC-derived models. Mol Therapy 2021 Jan 9;S1525-0016(21)00001-0.
- Anna Yang, Boris Kantor, and Ornit Chiba-Falek APOE: The New Frontier in the Development of a Therapeutic Target towards Precision Medicine in Late-Onset Alzheimer’s Disease. Int J of Mol Sci 2021 Life Sci Soc Policy 2020 Oct 12;16(1):11. doi: 10.1186/s40504-020-00106-2
- Gu, G, Barrera, J, Yun, Y, Murphy, S, Kantor, B and Chiba-Falek, O. Cell-type specific changes in DNA methylation of SNCA Intron1 in synucleinopathy brains Front Neurosci. 2021 Apr 28;15:652226. doi: 10.3389/fnins.2021.652226. eCollection 2021.
- Brown, LY, Dong, W; Kantor, B. An Improved Protocol for the Production of Lentiviral Vectors (2020) STAR Protocols 2020 Oct 27;1(3):100152.doi: 10.1016/j.xpro.2020.100152. eCollection 2020 Dec 18.
- Rittiner J, Moncalvo M, Chiba-Falek O , Kantor B. Gene-editing technologies paired with viral vectors for advancing basic and translation research of Neurodegenerative Diseases. Mol. Neurosci., 12 August 2020 | https://doi.org/10.3389/fnmol.2020.00148
- Uchitel J, Kantor B, Smith E, Mikati M. Viral-mediated gene replacement therapy in the developing Central Nervous System: current status and future directions (2020) Pediatric Neurology Vol 110, Sept 2020, Pages 5-19 https://doi.org/10.1016/j.pediatrneurol.2020.04.010
- Chen V, Moncalvo M, Tringali D, Tagliafierro L, Shriskanda A, Ilich E, Dong W, Kantor B, Chiba-Falek O. The mechanistic role of alpha-synuclein in the nucleus: Impaired nuclear function caused by familial Parkinson’s Disease SNCA mutations (2020) Hum Mol Genet. 2020 Nov 4;29(18):3107-3121. doi: 10.1093/hmg/ddaa183.
- Angrist, M, Yang A, Kantor B, Chiba-Falek, O. Good problems to have? Policy and societal implications of a disease-modifying therapy for presymptomatic Late-Onset Alzheimer’s Disease (2020) Life Sciences, Society and Policy 2020 Oct 12;16(1):11. doi: 10.1186/s40504-020-00106-2.
- Tagliafierro L, Ilich E, Moncalvo M, Gu LG, Sriskanda A, Grenier C, Murphy S, Chiba-Falek O, Kantor B. Lentiviral vector platform for the efficient delivery of epigenome-editing tools into human induced pluripotent stem cell-derived disease models (2019). J Vis Exp. 2019 Mar 29; (145):10.3791/59241. doi: 10.3791/59241.
- Kantor B, Tagliafierro L, Gu, LG, Zamora ME, Ilich K, Grenier C, Huang ZY, Murphy S, Chiba-Falek O. Downregulation of SNCA expression by targeted editing of DNA-methylation: A potential strategy for precision therapy in PD. Molecular Therapy (2018). 2018 Nov 7;26(11):2638-2649. doi: 10.1016/j.ymthe.2018.08.019. Epub 2018 Aug 29.
- Vijayraghavan S and Kantor B. Production of Integrase-Deficient Lentiviral Vector for Highly Efficient CRISPR/Cas9-Mediated Gene Knockout in Dividing Cells (2017). ). J Vis Exp. 2017 Dec 12;(130):56915. doi: 10.3791/56915.
- Ortinski PI, O’Donovan B, Dong X, and Kantor B. Integrase-deficient lentiviral vector as an all-in-one platform for highly efficient CRISPR/Cas9-mediated gene editing. (2017). Molecular Therapy Methods & Clinical Development 2017 Apr 19;5:153-164. doi: 10.1016/j.omtm.2017.04.002. eCollection 2017 Jun 16.
- Manuel A, Piroli G, Martin S, Kantor B, Adam J, et al. Succination of Protein Disulfide Isomerase Links Mitochondrial Stress to Endoplasmic Reticulum Stress in the Adipocyte during Diabetes. Antioxidants and Redox Signaling. 2017 April 04; doi: 10.1089/ars.2016.6853. [Epub ahead of print].
- Kantor B, Lentz TB, Nagabhushan Kalburgi S, McCown TJ, and Gray, SJ. Advances in viral-mediated gene delivery (2014). Advances in Molecular Genetics; 2014; 87:125-97. doi: 10.1016/B978-0-12-800149-3.00003-2.2
- Kantor B, Nagabhushan Kalburgi S, McCown TJ, and Gray, SJ. Clinical Applications Involving CNS Gene Transfer (2014). Advances in Molecular Genetics; 2014;87:71-124. doi: 10.1016/B978-0-12-800149-3.00002-0
- Titus MA, Zeithaml B, Kantor B, Li X, Haack K, Moore DT, Wilson EM, Mohler1 JL, and Kafri T. Dominant-Negative Androgen Receptor Inhibition of Intracrine Androgen-Dependent Growth of Castration-Recurrent Prostate Cancer (2012). PLoS One. 2012; 7(1):e30192. Epub 2012 Jan 17.
- Johnson JS, Gentzsch M, Zhang L, Ribeiro CM, Kantor B, Kafri T, Pickles RJ, Samulski RJ. AAV exploits subcellular stress associated with inflammation, endoplasmic reticulum expansion, and misfolded proteins in models of cystic fibrosis. (2011). PLoS Pathog. 2011 May;7(5):e1002053. Epub 2011 May 19.
- Kantor B, Bayer M, Ma H, Samulski J, Li C, McCown T, Kafri T. Notable reduction in illegitimate integration mediated by a PPT-deleted, nonintegrating lentiviral vector. (2011). Mol Ther. 2011 Mar; 19(3):547-56. Epub 2010 Dec 14.
- Monahan PE, Lothrop CD, Sun J, Hirsch ML, Kafri T, Kantor B, Sarkar R, Tillson DM, Elia JR, Samulski RJ. (2010). Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application. Mol Ther. 18(11):1907-16. Epub Aug 10. 2010.
- Kantor B, Ma H, Webster-Cyriaque J, Monahan PE, Kafri T. Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection (2009). Proc Natl Acad Sci U S A. Nov 3; 106(44):18786-91.
- Bayer M, Kantor B, Cockrell A, Hong M, Zeithaml B, Li X, McCown, T, and Kafri T. A large U3 deletion causes increased in vivo expression from a nonintegrated lentiviral vector (2008). Molecular Therapy. Advance online publication 16 September 2008. doi: 10.1038/mt.2008.199
Media Releases
New CRISPR-based strategies for Alzheimer disease. Nature Review Neurology. 19, page 507 (2023). New CRISPR-based strategies for Alzheimer disease | Nature Reviews Neurology
https://www.nature.com/articles/s41582-023-00856-5
News; Neurology Live Jlu 28, 2023
Early Intervention and Disease Prevention With a Novel Epigenome Approach: Boris Kantor, PhD
Early Intervention and Disease Prevention With a Novel Epigenome Approach: Boris Kantor, PhD
News; Neurology Live. August 3, 2023
Innovative Epigenome Editing Adds to Precision Medicine Approach for Alzheimer Disease
News; Neurology Live. July 30, 2023
Epigenetic Therapy Demonstrates Efficacy in APOE Reduction for Alzheimer Disease
Epigenetic Therapy Demonstrates Efficacy in APOE Reduction for Alzheimer Disease
Boris Kantor, Ph.D. | Science | AAAS
News; Practical Neurology April 24, 2024
CRISPR/dCAS9 Shows Promise in Editing APOE ε4 to Treat Late Onset Alzheimer Disease
Patent Applications
1. DEMENTIA WITH LEWY BODY: CELL SPECIFIC SNCA-TARGETED GENE THERAPY T-007901 Provisional Pending Conversion
2. DEVELOPMENT OF SELF-ACTIVATED VIRAL VECTORS T-007835 Provisional Pending Conversion
3. DOWNREGULATION OF HISTONE DEACETYLASE (HDAC) REPRESSORS AS AN EFFECTIVE MEANS OF IMPROVING LENTIVIRUS- TITER T-007108 Provisional Pending Conversion
4. PRE-PACKAGING OF CRISPR/CAS INTO VIRAL LIKE PARTICLES (VLP) FOR SAFER GENE THERAPY T-007523 Provisional Pending Conversion
5. A NOVEL ALL-IN-ONE AAV SYSTEM FOR EFFICIENT GENOME-EDITING APPROACH 63/256,754 STATUS: PCT CONVERTED
6. SYSTEMS AND METHODS OF IMPROVING LENTIVIRUS TITER 63/221,167 PCT CONVERTED, PUBLISHED
7. DOWNREGULATION OF APOE GENE EXPRESSION BY TARGETED EDITING OF DNA-METHYLATION AS THE APPROACH FOR TREATMENT OF ALZHEIMER’S DISEASE 63/132,286 PROVISIONAL STATUS: PCT CONVERTED, PUBLISHED
8. COMPOSITIONS AND METHODS FOR THE TREATMENT OF ATPASE-MEDIATED DISEASES PCT/US2020/032978 PCT CONVERTED, PUBLISHED
9. COMPOSITIONS AND METHODS RELATING TO ALZHEIMER’S DISEASE PCT/US2021/054475 PCT CONVERTED, NATIONAL PHASE / VALIDATION
10. DOWNREGULATION OF SNCA EXPRESSION BY TARGETED EDITING OF DNA-METHYLATION PCT/US2019/028786 STATUS: NATIONAL PHASE / VALIDATION (LICENSED TO SEELOS)
Email: viral.core@med.fsu.edu
Phone:(850) 644-4241
Mailing Address:
Boris Kantor, Ph.D.
Viral Vector and Gene Editing Core
Florida Institute of Pediatric and Rare Diseases
Florida State University
1115 West Call Street
Lab room 3310
Tallahassee, FL 32306