Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dys- trophy. Mol Ther 2013; 21(4): 750-7.Shin,J.H., Pan,X., Hakim,C.H., Yang,H.T., Yue,Y., Zhang,K., Terjung,R.L. and Duan,D. (2013) Microdystrophin Ameliorates Muscular Dystrophy in the ...
Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdxMice.Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdxMice.Dystrophin play...
In particular, the present invention provides can be recovered nitric oxide synthase nerve (nNOS) in the sarcolemma, the synthetic mini / micro-dystrophin gene set. Method is also provided with a pharmaceutical composition for the treatment of Duchenne muscular dystrophy (DMD), Becker muscular ...
Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The ...
Both approaches use microdystrophin proteins either directly as a desired construct for gene delivery, using the capacity-limited AAV vectors, or as the therapeutic outcome of gene splicing. Although functionality of the resulting artificial dystrophin proteins can be predicted in silico, experimental ...
The current optimised AAV-microdystrophin configuration thus lays a sound basis for a translation programme towards clinical trials in \\{DMD\\} patients.G.DicksonandC.LeGuinerandM.MontusandEBSCO_AspNeuromuscular DisordersDickson G, Le Guiner C, Montus M, et al. Perspec- tives on micro...