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ONE-STEP GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY VIA GENE REPLACEMENT AND ANTI-INFLAMMATION
ONE-STEP GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY VIA GENE REPLACEMENT AND ANTI-INFLAMMATION
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机译：通过基因替代和抗炎，杜氏肌营养不良的一步基因治疗
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摘要
In one embodiment, the invention provides a dual-cassette gene vehicle comprising cassettes for expression of both a mini-dystrophin gene and NF-κB/p65-shRNA gene in cardiac muscle tissue and skeletal muscle tissue, which is an adeno-associated viral (AAV) vector, wherein the mini-dystrophin gene is operably linked to a construct comprising a muscle-specific first promoter and a modified Mcken (MCK) enhancer and wherein the NF- κB/p65-shRNA gene is under the control of a second promoter. Also are provided pharmaceutical compositions comprising such gene vehicles and a method for ameliorating Duchenne muscular dystrophy (DMD) employing such gene delivery vehicles and pharmaceutical compositions.
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