Article
Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2014
Melo Sandra P, Lisowski Leszek, Bashkirova Elizaveta, Zhen Hanson H, Chu Kirk, Keene Douglas R, Marinkovich M Peter, Kay Mark A, Oro Anthony E
Abstract excerpt
Definitive correction of disease causing mutations in somatic cells by homologous recombination (HR) is an attractive therapeutic approach for the treatment of genetic diseases. However, HR-based somatic gene therapy is limited by the low efficiency of gene targeting in mammalian cells and replicative senescence of primary cells ex vivo, forcing investigators to explore alternative strategies such as retro- and...
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