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Check out our new publications focused on the development and optimization of viral vector tools paired with CRISPR/Cas9 systems for treatment of neurodegenerative diseases and disorders.
1. O’Donovan B, Rittiner J, Upadhya S, Hodgson D, Kantor B and Chiba-Falek O. Trends and challenges of AAV-delivered gene editing therapeutics for unmet needs in CNS disorders: Implications for neurodegenerative disease. Molecular Therapy - Nucleic Acids 2025 Accepted for publication.
2. Chiba-Falek O, Moncalvo M, Johnson, B, Lindner N, and Kantor B. A novel bimodal lentiviral vector-based inducible platform for modeling of Parkinson’s disease. Viruses 2025 (accepted for publication)
3. Rittiner J and Kantor B. Progress in Neuromethods Springer Nature. Book Chapter Gene to cell transfer into the CNS 2025 (accepted for publication).
4. 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.
5. 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.
6. 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.
7. 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 Front. Bioeng. Biotechnol., 2022 Sec. Preclinical Cell and Gene Therapy https://doi.org/10.3389/fbioe.2022.1035543
8. 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).
9. 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
10. 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
11. 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.
12. 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
13. 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.
14. 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.
15. 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. Front. Mol. Neurosci., 12 August 2020 | https://doi.org/10.3389/fnmol.2020.00148
16. 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
17. 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.
18. 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.
19. 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.
20. 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.
21. 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.
22. 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: APOE-targeted therapy for precision medicine in late onset Alzheimer's disease: A novel epigenome editing approach for downregulation of APOEe4 expression
New 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)