Developing AAV-based gene therapy for adrenoleukodystrophy (X-ALD)
Sign Up to like & getrecommendations! Published in 2019 at "Cytotherapy"
DOI: 10.1016/j.jcyt.2019.04.046
Abstract: Background & Aim Adrenoleukodystrophy (X-ALD) is an X-linked disease. ALD is caused by mutations of the gene encoding ATP-Binding cassette subfamily D member 1 (ABCD1), a peroxisomal transmembrane protein. Dysfunctional ABCD1 protein results in excessive… read more here.
Keywords: therapy; ald; based gene; aav based ... See more keywords
AAV-based gene therapies for the muscular dystrophies.
Sign Up to like & getrecommendations! Published in 2019 at "Human molecular genetics"
DOI: 10.1093/hmg/ddz128
Abstract: Muscular dystrophy is a group of progressive genetic diseases affecting the musculature which are characterized by inflammatory infiltrates, necrosis and connective tissue and fat replacement of the affected muscles. Unfortunately, treatments do not exist for… read more here.
Keywords: muscular dystrophies; muscular dystrophy; dystrophy; based gene ... See more keywords