Gene therapy for the hemoglobin disorders: Past, present, and future

被引:32
作者
Persons, DA [1 ]
Nienhuis, AW [1 ]
机构
[1] St Jude Childrens Res Hosp, Dept Hematol & Oncol, Div Expt Hematol, Memphis, TN 38105 USA
关键词
D O I
10.1073/pnas.97.10.5022
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
引用
收藏
页码:5022 / 5024
页数:3
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共 40 条
[11]   LINEAGE-SPECIFIC EXPRESSION OF A HUMAN BETA-GLOBIN GENE IN MURINE BONE-MARROW TRANSPLANT RECIPIENTS RECONSTITUTED WITH RETROVIRUS-TRANSDUCED STEM-CELLS [J].
DZIERZAK, EA ;
PAPAYANNOPOULOU, T ;
MULLIGAN, RC .
NATURE, 1988, 331 (6151) :35-41
[12]   Analysis of γ-globin expression cassettes in retrovirus vectors [J].
Emery, DW ;
Morrish, F ;
Li, QL ;
Stamatoyannopoulos, G .
HUMAN GENE THERAPY, 1999, 10 (06) :877-888
[13]   THE HUMAN BETA-GLOBIN LOCUS ACTIVATION REGION ALTERS THE DEVELOPMENTAL FATE OF A HUMAN-FETAL GLOBIN GENE IN TRANSGENIC MICE [J].
ENVER, T ;
EBENS, AJ ;
FORRESTER, WC ;
STAMATOYANNOPOULOS, G .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1989, 86 (18) :7033-7037
[14]   The β-globin LCR is not necessary for an open chromatin structure or developmentally regulated transcription of the native mouse β-globin locus [J].
Epner, E ;
Reik, A ;
Cimbora, D ;
Telling, A ;
Bender, MA ;
Fiering, S ;
Enver, T ;
Martin, DIK ;
Kennedy, M ;
Keller, G ;
Groudine, M .
MOLECULAR CELL, 1998, 2 (04) :447-455
[15]   EVIDENCE FOR A LOCUS ACTIVATION REGION - THE FORMATION OF DEVELOPMENTALLY STABLE HYPERSENSITIVE SITES IN GLOBIN-EXPRESSING HYBRIDS [J].
FORRESTER, WC ;
TAKEGAWA, S ;
PAPAYANNOPOULOU, T ;
STAMATOYANNOPOULOS, G ;
GROUDINE, M .
NUCLEIC ACIDS RESEARCH, 1987, 15 (24) :10159-10177
[16]   EMBRYONIC STEM-CELL VIRUS, A RECOMBINANT MURINE RETROVIRUS WITH EXPRESSION IN EMBRYONIC STEM-CELLS [J].
GREZ, M ;
AKGUN, E ;
HILBERG, F ;
OSTERTAG, W .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1990, 87 (23) :9202-9206
[17]   POSITION-INDEPENDENT, HIGH-LEVEL EXPRESSION OF THE HUMAN BETA-GLOBIN GENE IN TRANSGENIC MICE [J].
GROSVELD, F ;
VANASSENDELFT, GB ;
GREAVES, DR ;
KOLLIAS, G .
CELL, 1987, 51 (06) :975-985
[18]   Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells [J].
Hanenberg, H ;
Xiao, XL ;
Dilloo, D ;
Hashino, K ;
Kato, I ;
Williams, DA .
NATURE MEDICINE, 1996, 2 (08) :876-882
[19]   Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human β-globin in engrafted mice [J].
Kalberer, CP ;
Pawliuk, R ;
Imren, S ;
Bachelot, T ;
Takekoshi, KJ ;
Fabry, M ;
Eaves, CJ ;
London, IM ;
Humphries, RK ;
Leboulch, P .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2000, 97 (10) :5411-5415
[20]   EXPRESSION OF THE HUMAN BETA-GLOBIN GENE FOLLOWING RETROVIRAL-MEDIATED TRANSFER INTO MULTIPOTENTIAL HEMATOPOIETIC PROGENITORS OF MICE [J].
KARLSSON, S ;
BODINE, DM ;
PERRY, L ;
PAPAYANNOPOULOU, T ;
NIENHUIS, AW .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1988, 85 (16) :6062-6066