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 Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

95 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Base editing as a genetic treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. Among authors: maguire ca. bioRxiv [Preprint]. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. bioRxiv. 2023. PMID: 36711797 Free PMC article. Preprint.
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease.
György B, Lööv C, Zaborowski MP, Takeda S, Kleinstiver BP, Commins C, Kastanenka K, Mu D, Volak A, Giedraitis V, Lannfelt L, Maguire CA, Joung JK, Hyman BT, Breakefield XO, Ingelsson M. György B, et al. Among authors: maguire ca. Mol Ther Nucleic Acids. 2018 Jun 1;11:429-440. doi: 10.1016/j.omtn.2018.03.007. Epub 2018 Mar 16. Mol Ther Nucleic Acids. 2018. PMID: 29858078 Free PMC article.
High levels of AAV vector integration into CRISPR-induced DNA breaks.
Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Lööv C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, György B. Hanlon KS, et al. Among authors: maguire ca. Nat Commun. 2019 Sep 30;10(1):4439. doi: 10.1038/s41467-019-12449-2. Nat Commun. 2019. PMID: 31570731 Free PMC article.
In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord.
Hanlon KS, Cheng M, De La Cruz D, Patel N, Santoscoy MC, Gong Y, Ng C, Nguyen DM, Nammour J, Clark SW, Kozarsky K, Maguire CA. Hanlon KS, et al. Among authors: maguire ca. bioRxiv [Preprint]. 2023 Sep 13:2023.09.13.557506. doi: 10.1101/2023.09.13.557506. bioRxiv. 2023. Update in: Mol Ther. 2024 Aug 7;32(8):2584-2603. doi: 10.1016/j.ymthe.2024.05.040 PMID: 37745398 Free PMC article. Updated. Preprint.
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord.
Hanlon KS, Cheng M, Ferrer RM, Ryu JR, Lee B, De La Cruz D, Patel N, Espinoza P, Santoscoy MC, Gong Y, Ng C, Nguyen DM, Nammour J, Clark SW, Heine VM, Sun W, Kozarsky K, Maguire CA. Hanlon KS, et al. Among authors: maguire ca. Mol Ther. 2024 Aug 7;32(8):2584-2603. doi: 10.1016/j.ymthe.2024.05.040. Epub 2024 Jun 5. Mol Ther. 2024. PMID: 38845196 Free PMC article.
Metabolic engineering improves transduction efficiency and downstream vector isolation by altering the lipid composition of extracellular vesicle-enclosed AAV.
Espinoza P, Cheng M, Ng C, De La Cruz D, Wasson ED, McCarthy DM, Bhide PG, Maguire CA, Santoscoy MC. Espinoza P, et al. Among authors: maguire ca. bioRxiv [Preprint]. 2024 Aug 19:2024.08.16.608303. doi: 10.1101/2024.08.16.608303. bioRxiv. 2024. Update in: Metab Eng. 2024 Dec 7;88:40-49. doi: 10.1016/j.ymben.2024.12.003 PMID: 39229172 Free PMC article. Updated. Preprint.
Expression-based selection identifies a microglia-tropic AAV capsid for direct and CSF routes of administration in mice.
Santoscoy MC, Espinoza P, Hanlon KS, Yang L, Nieland L, Ng C, Badr CE, Hickman S, de la Cruz D, Griciuc A, Elkhoury J, Bennett RE, Shen S, Maguire CA. Santoscoy MC, et al. Among authors: maguire ca. bioRxiv [Preprint]. 2024 Sep 27:2024.09.25.614546. doi: 10.1101/2024.09.25.614546. bioRxiv. 2024. PMID: 39386560 Free PMC article. Preprint.
CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice.
Nieland L, Vrijmoet AB, Jetten IW, Rufino-Ramos D, de Reus AJEM, Breyne K, Kleinstiver BP, Maguire CA, Broekman MLD, Breakefield XO, Abels ER. Nieland L, et al. Among authors: maguire ca. Mol Ther. 2024 Nov 19:S1525-0016(24)00750-0. doi: 10.1016/j.ymthe.2024.11.023. Online ahead of print. Mol Ther. 2024. PMID: 39563028 Free article.
95 results