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

225 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-mediated splicing correction therapy for spinal muscular atrophy.
Lin X, Chen H, Lu YQ, Hong S, Hu X, Gao Y, Lai LL, Li JJ, Wang Z, Ying W, Ma L, Wang N, Zuo E, Yang H, Chen WJ. Lin X, et al. Cell Res. 2020 Jun;30(6):548-550. doi: 10.1038/s41422-020-0304-y. Epub 2020 Mar 24. Cell Res. 2020. PMID: 32210360 Free PMC article. No abstract available.
Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice.
Li JJ, Lin X, Tang C, Lu YQ, Hu X, Zuo E, Li H, Ying W, Sun Y, Lai LL, Chen HZ, Guo XX, Zhang QJ, Wu S, Zhou C, Shen X, Wang Q, Lin MT, Ma LX, Wang N, Krainer AR, Shi L, Yang H, Chen WJ. Li JJ, et al. Among authors: ma lx. Natl Sci Rev. 2019 Sep 3;7(1):92-101. doi: 10.1093/nsr/nwz131. eCollection 2020 Jan. Natl Sci Rev. 2019. PMID: 34691481 Free PMC article.
225 results