Article
Gene targeting of mutant COL1A2 alleles in mesenchymal stem cells from individuals with osteogenesis imperfecta.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jan 2008
Chamberlain Joel R, Deyle David R, Schwarze Ulrike, Wang Peirong, Hirata Roli K, Li Yi, Byers Peter H, Russell David W
Abstract excerpt
Mesenchymal stem cells (MSCs) are adult cells with the capacity to differentiate into multiple cell types, including bone, fat, cartilage, and muscle cells. In order to effectively utilize autologous MSCs in cell-based therapies, precise genetic manipulations are required to eliminate the effects of disease-causing mutations. We previously used adeno-associated virus (AAV) vectors to target and inactivate mutant...
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