Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

203 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Rescue of glutaric aciduria type I in mice by liver-directed therapies.
Barzi M, Johnson CG, Chen T, Rodriguiz RM, Hemmingsen M, Gonzalez TJ, Rosales A, Beasley J, Peck CK, Ma Y, Stiles AR, Wood TC, Maeso-Diaz R, Diehl AM, Young SP, Everitt JI, Wetsel WC, Lagor WR, Bissig-Choisat B, Asokan A, El-Gharbawy A, Bissig KD. Barzi M, et al. Among authors: asokan a. Sci Transl Med. 2023 Apr 19;15(692):eadf4086. doi: 10.1126/scitranslmed.adf4086. Epub 2023 Apr 19. Sci Transl Med. 2023. PMID: 37075130 Free PMC article.
Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery.
Gonzalez TJ, Mitchell-Dick A, Blondel LO, Fanous MM, Hull JA, Oh DK, Moller-Tank S, Castellanos Rivera RM, Piedrahita JA, Asokan A. Gonzalez TJ, et al. Among authors: asokan a. Nat Protoc. 2023 Nov;18(11):3413-3459. doi: 10.1038/s41596-023-00875-y. Epub 2023 Sep 21. Nat Protoc. 2023. PMID: 37735235 Review.
A humanized mouse model for adeno-associated viral gene therapy.
Barzi M, Chen T, Gonzalez TJ, Pankowicz FP, Oh SH, Streff HL, Rosales A, Ma Y, Collias S, Woodfield SE, Diehl AM, Vasudevan SA, Galvan TN, Goss J, Gersbach CA, Bissig-Choisat B, Asokan A, Bissig KD. Barzi M, et al. Among authors: asokan a. Nat Commun. 2024 Mar 4;15(1):1955. doi: 10.1038/s41467-024-46017-0. Nat Commun. 2024. PMID: 38438373 Free PMC article.
Repurposing CRISPR-Cas13 systems for robust mRNA trans-splicing.
Fiflis DN, Rey NA, Venugopal-Lavanya H, Sewell B, Mitchell-Dick A, Clements KN, Milo S, Benkert AR, Rosales A, Fergione S, Asokan A. Fiflis DN, et al. Among authors: asokan a. Nat Commun. 2024 Mar 14;15(1):2325. doi: 10.1038/s41467-024-46172-4. Nat Commun. 2024. PMID: 38485709 Free PMC article.
The NIH Somatic Cell Genome Editing program.
Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB Jr, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J; SCGE Consortium. Saha K, et al. Among authors: asokan a. Nature. 2021 Apr;592(7853):195-204. doi: 10.1038/s41586-021-03191-1. Epub 2021 Apr 7. Nature. 2021. PMID: 33828315 Free PMC article. Review.
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
Nelson CE, Wu Y, Gemberling MP, Oliver ML, Waller MA, Bohning JD, Robinson-Hamm JN, Bulaklak K, Castellanos Rivera RM, Collier JH, Asokan A, Gersbach CA. Nelson CE, et al. Among authors: asokan a. Nat Med. 2019 Mar;25(3):427-432. doi: 10.1038/s41591-019-0344-3. Epub 2019 Feb 18. Nat Med. 2019. PMID: 30778238 Free PMC article.
203 results