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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.
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. Among authors: kaspar bk, kaspar a. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Free article. Clinical Trial.
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.
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.
116 results