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

2,912 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. Harmatz P, et al. Among authors: meyer k. Mol Ther. 2022 Dec 7;30(12):3587-3600. doi: 10.1016/j.ymthe.2022.10.010. Epub 2022 Oct 25. Mol Ther. 2022. PMID: 36299240 Free PMC article.
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
Laoharawee K, DeKelver RC, Podetz-Pedersen KM, Rohde M, Sproul S, Nguyen HO, Nguyen T, St Martin SJ, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T, McIvor RS. Laoharawee K, et al. Among authors: meyer ke. Mol Ther. 2018 Apr 4;26(4):1127-1136. doi: 10.1016/j.ymthe.2018.03.002. Epub 2018 Mar 10. Mol Ther. 2018. PMID: 29580682 Free PMC article.
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Ou L, DeKelver RC, Rohde M, Tom S, Radeke R, St Martin SJ, Santiago Y, Sproul S, Przybilla MJ, Koniar BL, Podetz-Pedersen KM, Laoharawee K, Cooksley RD, Meyer KE, Holmes MC, McIvor RS, Wechsler T, Whitley CB. Ou L, et al. Among authors: meyer ke. Mol Ther. 2019 Jan 2;27(1):178-187. doi: 10.1016/j.ymthe.2018.10.018. Epub 2018 Nov 1. Mol Ther. 2019. PMID: 30528089 Free PMC article.
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Yasuda M, Huston MW, Pagant S, Gan L, St Martin S, Sproul S, Richards D, Ballaron S, Hettini K, Ledeboer A, Falese L, Cao L, Lu Y, Holmes MC, Meyer K, Desnick RJ, Wechsler T. Yasuda M, et al. Among authors: meyer k. Mol Ther Methods Clin Dev. 2020 Jul 9;18:607-619. doi: 10.1016/j.omtm.2020.07.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775495 Free PMC article.
A robust and flexible baculovirus-insect cell system for AAV vector production with improved yield, capsid ratios and potency.
Marwidi Y, Nguyen HB, Santos D, Wangzor T, Bhardwaj S, Ernie G, Prawdzik G, Lew G, Shivak D, Trias M, Padilla J, Tran H, Meyer K, Surosky R, Ward AM. Marwidi Y, et al. Among authors: meyer k. Mol Ther Methods Clin Dev. 2024 Mar 4;32(2):101228. doi: 10.1016/j.omtm.2024.101228. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38524756 Free PMC article.
Gene Editing: Regulatory and Translation to Clinic.
Ando D, Meyer K. Ando D, et al. Among authors: meyer k. Hematol Oncol Clin North Am. 2017 Oct;31(5):797-808. doi: 10.1016/j.hoc.2017.06.002. Hematol Oncol Clin North Am. 2017. PMID: 28895848 Review.
2,912 results