Gene therapy: Trials and tribulations

被引：518

作者：

Somia, N ^{[1
]}

Verma, IM ^{[1
]}

机构：

[1] Salk Inst Biol Studies, Genet Lab, La Jolla, CA 92037 USA

来源：

NATURE REVIEWS GENETICS | 2000年 / 1卷 / 02期

关键词：

D O I：

10.1038/35038533

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

The art and science of gene therapy has received much attention of late. The tragic death of 18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the successful treatment of three children suffering from a rare but fatal immunological disease. In the light of the success and tragedy, it is timely to consider the challenges faced by gene therapy - a novel form of molecular medicine that may be poised to have an important impact on human health in the new millennium.

引用

页码：91 / 99

页数：9

共 77 条

[41] HUMAN GENE-THERAPY COMES OF AGE
MILLER, AD
[J]. NATURE, 1992, 357 (6378) : 455 - 460
[42] Stable gene transter to the nervous system using a non-primate lentiviral vector
Mitrophanous, KA
Yoon, S
Rohll, JB
Patil, D
Wilkes, FJ
Kim, VN
Kingsman, SM
Kingsman, AJ
Mazarakis, ND
[J]. GENE THERAPY, 1999, 6 (11) : 1808 - 1818
[43] Development of a self-inactivating lentivirus vector
Miyoshi, H
Blömer, U
Takahashi, M
Gage, FH
Verma, IM
[J]. JOURNAL OF VIROLOGY, 1998, 72 (10) : 8150 - 8157
[44] Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
Miyoshi, H
Smith, KA
Mosier, DE
Verma, IM
Torbett, BE
[J]. SCIENCE, 1999, 283 (5402) : 682 - 686
[45] Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector
Miyoshi, H
Takahashi, M
Gage, FH
Verma, IM
[J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (19) : 10319 - 10323
[46] MORGAN RA, 1993, ANNU REV BIOCHEM, V62, P191
[47] Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
Morral, N
O'Neal, W
Rice, K
Leland, M
Kaplan, J
Piedra, PA
Zhou, HS
Parks, RJ
Velji, R
Aguilar-Córdova, E
Wadsworth, S
Graham, FL
Kochanek, S
Carey, KD
Beaudet, AL
[J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1999, 96 (22) : 12816 - 12821
[48] MULIGAN RC, 1993, SCIENCE, V260, P926
[49] Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors
Nakai, H
Storm, TA
Kay, MA
[J]. NATURE BIOTECHNOLOGY, 2000, 18 (05) : 527 - 532
[50] In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
Naldini, L
Blomer, U
Gallay, P
Ory, D
Mulligan, R
Gage, FH
Verma, IM
Trono, D
[J]. SCIENCE, 1996, 272 (5259) : 263 - 267

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