Ontology highlight
ABSTRACT:
SUBMITTER: Chamberlain JR
PROVIDER: S-EPMC4128632 | biostudies-literature | 2008 Jan
REPOSITORIES: biostudies-literature
Chamberlain Joel R JR Deyle David R DR Schwarze Ulrike U Wang Peirong P Hirata Roli K RK Li Yi Y Byers Peter H PH Russell David W DW
Molecular therapy : the journal of the American Society of Gene Therapy 20071023 1
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 COL1A1 genes in MSCs from individuals with the brittle bone disorder, osteogenesis i ...[more]