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

116 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, Lewis S, McColly M, Hogan MJ, Kaspar BK. Mendell JR, et al. Among authors: kaspar bk. Mol Ther. 2017 Apr 5;25(4):870-879. doi: 10.1016/j.ymthe.2017.02.015. Epub 2017 Mar 6. Mol Ther. 2017. PMID: 28279643 Free PMC article.
Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.
Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Kota J, et al. Among authors: kaspar bk. Sci Transl Med. 2009 Nov 11;1(6):6ra15. doi: 10.1126/scitranslmed.3000112. Sci Transl Med. 2009. PMID: 20368179 Free PMC article.
Gene therapy for muscular dystrophy: lessons learned and path forward.
Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR. Mendell JR, et al. Among authors: kaspar bk. Neurosci Lett. 2012 Oct 11;527(2):90-9. doi: 10.1016/j.neulet.2012.04.078. Epub 2012 May 17. Neurosci Lett. 2012. PMID: 22609847 Free PMC article. Review.
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK. Mendell JR, et al. Among authors: kaspar bk. Mol Ther. 2015 Jan;23(1):192-201. doi: 10.1038/mt.2014.200. Epub 2014 Oct 17. Mol Ther. 2015. PMID: 25322757 Free PMC article. Clinical Trial.
Could gene therapy be the future for muscular dystrophy?
Haidet AM, Mendell JR, Kaspar BK. Haidet AM, et al. Among authors: kaspar bk. Therapy. 2010 May 1;7(3):287-290. doi: 10.2217/thy.10.14. Therapy. 2010. PMID: 20543898 Free PMC article. No abstract available.
AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.
Sahenk Z, Galloway G, Clark KR, Malik V, Rodino-Klapac LR, Kaspar BK, Chen L, Braganza C, Montgomery C, Mendell JR. Sahenk Z, et al. Among authors: kaspar bk. Mol Ther. 2014 Mar;22(3):511-521. doi: 10.1038/mt.2013.250. Epub 2013 Oct 28. Mol Ther. 2014. PMID: 24162799 Free PMC article.
116 results