VIRAL VECTOR & GENE EDITING CORE
Media
Check out our publicationsfocused on the development and optimization of viral vector tools paired with CRISPR/Cas9 systems for treatment of genetic diseases.
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
- 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
Innovative Epigenome Editing Adds to Precision Medicine Approach for Alzheimer Disease
Epigenetic Therapy Demonstrates Efficacy in APOE Reduction for Alzheimer Disease
Early Intervention and Disease Prevention With a Novel Epigenome Approach
New CRISPR-based strategies for Alzheimer disease
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)