Publication Date:
2004-02-21
Description:
Adult stem cells offer the potential to treat many diseases through a combination of ex vivo genetic manipulation and autologous transplantation. Mesenchymal stem cells (MSCs, also referred to as marrow stromal cells) are adult stem cells that can be isolated as proliferating, adherent cells from bones. MSCs can differentiate into multiple cell types present in several tissues, including bone, fat, cartilage, and muscle, making them ideal candidates for a variety of cell-based therapies. Here, we have used adeno-associated virus vectors to disrupt dominant-negative mutant COL1A1 collagen genes in MSCs from individuals with the brittle bone disorder osteogenesis imperfecta, demonstrating successful gene targeting in adult human stem cells.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Chamberlain, Joel R -- Schwarze, Ulrike -- Wang, Pei-Rong -- Hirata, Roli K -- Hankenson, Kurt D -- Pace, James M -- Underwood, Robert A -- Song, Kit M -- Sussman, Michael -- Byers, Peter H -- Russell, David W -- New York, N.Y. -- Science. 2004 Feb 20;303(5661):1198-201.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Department of Medicine, University of Washington, Seattle, WA 98195-7720, USA.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/14976317" target="_blank"〉PubMed〈/a〉
Keywords:
Alleles
;
Animals
;
Bone Marrow Cells/physiology
;
Cell Differentiation
;
Cells, Cultured
;
Collagen Type I/chemistry/*genetics/metabolism
;
Dependovirus/genetics
;
*Gene Targeting
;
Genetic Therapy
;
Genetic Vectors
;
Humans
;
Kanamycin Kinase/genetics
;
Male
;
Mesenchymal Stromal Cells/*physiology
;
Mice
;
Osteogenesis
;
Osteogenesis Imperfecta/*genetics/*therapy
;
Point Mutation
;
Recombination, Genetic
;
Stem Cell Transplantation
Print ISSN:
0036-8075
Electronic ISSN:
1095-9203
Topics:
Biology
,
Chemistry and Pharmacology
,
Computer Science
,
Medicine
,
Natural Sciences in General
,
Physics
