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Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251.
Ann Neurol. 2010.
PMID: 21031578
Free PMC article.
Clinical Trial.
INTERPRETATION: The findings of this gene replacement study in LGMD2D subjects have important implications not previously demonstrated in muscular dystrophy. Long-term, sustainable gene expression of alpha-sarcoglycan was observed following gene transfer mediated by AAV. . …
INTERPRETATION: The findings of this gene replacement study in LGMD2D subjects have important implications not previously demonstrated in mu …
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.
Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR.
Malik V, et al. Among authors: shilling cj.
Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024.
Ann Neurol. 2010.
PMID: 20517938
Clinical Trial.
OBJECTIVE: The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length dystrophin. ...
OBJECTIVE: The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthro …
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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: shilling cj.
Sci Transl Med. 2009 Nov 11;1(6):6ra15. doi: 10.1126/scitranslmed.3000112.
Sci Transl Med. 2009.
PMID: 20368179
Free PMC article.
When injected into the quadriceps of cynomolgus macaque monkeys, a follistatin isoform expressed from an adeno-associated virus serotype 1 vector, AAV1-FS344, induced pronounced and durable increases in muscle size and strength. Long-term expression of the transgene did no …
When injected into the quadriceps of cynomolgus macaque monkeys, a follistatin isoform expressed from an adeno-associated virus serotype 1 v …
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Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors.
Haidet AM, Rizo L, Handy C, Umapathi P, Eagle A, Shilling C, Boue D, Martin PT, Sahenk Z, Mendell JR, Kaspar BK.
Haidet AM, et al. Among authors: shilling c.
Proc Natl Acad Sci U S A. 2008 Mar 18;105(11):4318-22. doi: 10.1073/pnas.0709144105. Epub 2008 Mar 11.
Proc Natl Acad Sci U S A. 2008.
PMID: 18334646
Free PMC article.
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