Recent Publications

CNS targeted AAV gene therapies: Recent advances and clinical insights.

Brown, B.L., Lewis, R.W., and Davidson, B.L.

Cell Rep Med 2026;7(11):103118. doi: 10.1016/j.xcrm.2026.103118. (Full text here)

Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery

Ahrens-Nicklas RC, Kotch C, Roche AM, Everett JK, Reddy S, Santi M, Madsen PJ, Martos-Rus C, Yrigollen CM, Small JC, McCague S, Davidson BL, Samelson-Jones BJ, Surrey LF, Li M, Ficicioglu C, Wang R, Bushman FD, George LA.

N Engl J Med. 2026 Jun 4;394(21):2126-2133. doi: 10.1056/NEJMoa2601608. Epub 2026 May 13. PMID: 42127296.

Temporal single-cell atlas of full-length Huntington's disease mouse model defines stage-specific signatures of corticostriatal dysfunction

Ashley B. Robbins, Paul T. Ranum, Icnelia Huerto-Ocampo, Michael Kuckyr, Beverly L. Davidson

‍Mol Neurodegener. 2026 May 28;21(1):41. doi: 10.1186/s13024-026-00960-2. PMID: 42210302; PMCID: PMC13440178. ‍DOI: https://doi.org/10.64898/2026.01.07.698249

Computational modeling and preclinical validation support targeting somatic instability for Huntington’s disease treatment

Bryan P. Simpson, Paul T. Ranum, David E. Leib, Luis Tecedor, Ryan C. Giovenco, Icnelia Huerta-Ocampo, Amiel R. Hundley, Noah Smith, Christopher M. Fluta, Christopher P. Cali, Jamie Benoit, Jenna C. Soper, John Connelly, George J. Yohrling, P. Peter Ghoroghchian, Jang-Ho J. Cha, Beverly L. Davidson

‍Nat Commun 17, 11036 (28 Oct 2026). https://doi.org/10.1038/s41467-026-78769-2 ‍

AAV-based delivery of RNAi targeting ataxin2 improves survival and pathology in TDP43 mice

Defne A. Amado, Ashley B. Robbins, Katherine R. Whiteman, Alicia R. Smith, Guillem Chillon, Yonghong Chen, Joshua A. Fuller, Nicholas A. Patty, Aleksandar Izda, Congsheng Cheng, Shareen Nelson,Abigail I. Dichter , Esteban O. Mazzoni, Alex Mas Monteys, Beverly L. Davidson Nat. Comm. 2025 June 25

DOI: https://doi.org/10.1038/s41467-025-60497-8

Optimized AAV capsids for diseases of the basal ganglia show robust potency and distribution in adult nonhuman primates

David E. Leib, Yonghong Chen, Luis Tecedor, Paul T. Ranum, Megan S. Keiser, Brian C. Lewandowski, Ellie M. Carrell, Sakshi Arora, Icnelia Huerta Ocampo, Xueyuan Liu, Beverly L Davidson Nat. Comm. 2025 May 19

DOI: https://doi.org/10.1038/s41467-025-60000-3

Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder

Felix AJ, Brown BL, Marotta N, Gessner MJ, Houserova M, Huerta-Ocampo I, Wilson T, Randell R, Dawicki-McKenna JM, Reinhardt D, Uchida K, McSalley I, McKee JL, Helbig I, Boland MJ, Davidson BL, Prosser BL.

Mol Psychiatry. 2026 Jul 14. doi: 10.1038/s41380-026-03698-x. Epub ahead of print. PMID: 42448791.

Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD

Samelson-Jones BJ, Martos-Rus C, Yrigollen CM, Tecedor L, Small JC, Matesanz SE, Watson CT, Vanden Heuvel AR, Carrig S, Yum SW, Brandsema JF, Lin K, Wittlieb-Weber C, Viaene AN, Russo PP, Partington S, Huynh K, Seneviratne T, Juarez Rojas S, Doshi BS, Shieh P, Flanigan KM, Frair E, Nicolau S, Lawlor MW, Crudele JM, Davidson BL, George LA.

N Engl J Med. 2026 Aug 6;395(6):615-617. doi: 10.1056/NEJMc2518477. Epub 2026 Jul 22. PMID: 42485644.

Optimized AAV capsids robustly transduce airway epithelial cells

Cooney AL, Chen YH, Lewandowski BC, Lamer S, Boysen G, Kulhankova K, Vu A, Newase P, Sinn PL, Davidson BL, McCray PB Jr.

bioRxiv [Preprint]. 2026 May 11:2026.05.10.723853. doi: 10.64898/2026.05.10.723853. PMID: 42182483; PMCID: PMC13193003.

Co-Opting MBNL-Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy

Carrell ST, Carrell EM, Giovenco R, Davidson BL.

Ann Neurol. 2026 Jan;99(1):211-222. doi: 10.1002/ana.78024. Epub 2025 Aug 29. PMID: 40879030; PMCID: PMC12946605.

Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform

Feierman ER, Whittaker MN, Quigley A, Brooks DL, McVeigh P, Nan AX, Hsu A, Said H, Soliman OY, Giovenco R, Davidson BL, Alameh MG, Liu DR, Wang X, Musunuru K, Ahrens-Nicklas RC.

Am J Hum Genet. 2026 May 7;113(5):891-899. doi: 10.1016/j.ajhg.2026.03.018. Epub 2026 Mar 31. PMID: 41923647; PMCID: PMC13058825.

CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability

Sichlinger L, Reilly MB, Arora S, Zhang S, Marotta N, Rodríguez-Acevedo KL, Hooks M, Czarnecki KS, Winter JJ, Waxman EA, Dungan LV, Hong I, Araki Y, Johnson R, Huganir RL, Pavani G, French DL, Davidson BL, Prosser BL, Heller EA.

bioRxiv [Preprint]. 2025 Oct 29:2025.10.28.685100. doi: 10.1101/2025.10.28.685100. PMID: 41279390; PMCID: PMC12636332.