Gene targeting in stem cells from individuals with osteogenesis imperfecta

被引：207

作者：

Chamberlain, JR

Schwarze, U

Wang, PR

Hirata, RK

Hankenson, KD

Pace, JM

Underwood, RA

Song, KM

Sussman, M

Byers, PH

Russell, DW ^{[1
]}

机构：

[1] Univ Washington, Dept Med, Seattle, WA 98195 USA

[2] Univ Washington, Dept Pathol, Seattle, WA 98195 USA

[3] Univ Washington, Dept Biochem, Seattle, WA 98195 USA

[4] Univ Michigan, Dept Orthopaed Surg, Ann Arbor, MI 48109 USA

[5] Univ Michigan, Unit Lab Anim Med, Ann Arbor, MI 48109 USA

[6] Childrens Hosp & Med Ctr, Seattle, WA 98105 USA

[7] Shriners Hosp Children, Portland, OR 97201 USA

来源：

SCIENCE | 2004年 / 303卷 / 5661期

关键词：

D O I：

10.1126/science.1088757

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

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.

引用

页码：1198 / 1201

页数：4

共 21 条

[1] BONADIO J, 1985, J BIOL CHEM, V260, P1734
[2] BYERS PH, 2000, METABOLIC MOL BASIS, P5241
[3] MESENCHYMAL STEM-CELLS
CAPLAN, AI
[J]. JOURNAL OF ORTHOPAEDIC RESEARCH, 1991, 9 (05) : 641 - 650
[4] CONSTANTINOU CD, 1990, AM J HUM GENET, V47, P670
[5] Edwards Matthew J., 1992, Human Mutation, V1, P47, DOI 10.1002/humu.1380010108
[6] FEDARKO NS, 1992, J BONE MINER RES, V7, P921
[7] LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
Hacein-Bey-Abina, S
Von Kalle, C
Schmidt, M
McCcormack, MP
Wulffraat, N
Leboulch, P
Lim, A
Osborne, CS
Pawliuk, R
Morillon, E
Sorensen, R
Forster, A
Fraser, P
Cohen, JI
de Saint Basile, G
Alexander, I
Wintergerst, U
Frebourg, T
Aurias, A
Stoppa-Lyonnet, D
Romana, S
Radford-Weiss, I
Gross, F
Valensi, F
Delabesse, E
Macintyre, E
Sigaux, F
Soulier, J
Leiva, LE
Wissler, M
Prinz, C
Rabbitts, TH
Le Deist, F
Fischer, A
Cavazzana-Calvo, M
[J]. SCIENCE, 2003, 302 (5644) : 415 - 419
[8] Targeted transgene insertion into human chromosomes by adeno-associated virus vectors
Hirata, R
Chamberlain, J
Dong, R
Russell, DW
[J]. NATURE BIOTECHNOLOGY, 2002, 20 (07) : 735 - 738
[9] Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta
Horwitz, EM
Prockop, DJ
Fitzpatrick, LA
Koo, WWK
Gordon, PL
Neel, M
Sussman, M
Orchard, P
Marx, JC
Pyeritz, RE
Brenner, MK
[J]. NATURE MEDICINE, 1999, 5 (03) : 309 - 313
[10] Isolated allogeneic bone marrow-derived mesenchymal cells engraft and stimulate growth in children with osteogenesis imperfecta: Implications for cell therapy of bone
Horwitz, EM
Gordon, PL
Koo, WKK
Marx, JC
Neel, MD
McNall, RY
Muul, L
Hofmann, T
[J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2002, 99 (13) : 8932 - 8937

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