Gene therapy of storage disorders by retroviral and lentiviral vectors

被引：26

作者：

Biffi, A

Naldini, L

机构：

[1] San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

[2] Univ Vita Salute San Raffaele, Ist Sci San Raffaele, I-20132 Milan, Italy

来源：

HUMAN GENE THERAPY | 2005年 / 16卷 / 10期

关键词：

D O I：

10.1089/hum.2005.16.1133

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

[No abstract available]

引用

页码：1133 / 1142

页数：10

共 105 条

[61] Bone marrow stem cell gene therapy of arylsulfatase A-deficient mice, using an arylsulfatase A mutant that is hypersecreted from retrovirally transduced donor-type cells [J].

Matzner, U ;

Schestag, F ;

Hartmann, D ;

Lüllmann-Rauch, R ;

D'Hooge, R ;

De Deyn, PP ;

Gieselmann, V .

HUMAN GENE THERAPY, 2001, 12 (09) :1021-1033

[62] Factors affecting long-term expression of a secreted transgene product after intravenous administration of a retroviral vector [J].

McCormack, JE ;

Edwards, W ;

Sensintaffer, J ;

Lillegren, L ;

Kozloski, M ;

Brumm, D ;

Karavodin, L ;

Jolly, DJ ;

Greengard, J .

MOLECULAR THERAPY, 2001, 3 (04) :516-525

[63] Stem cells in the central nervous system [J].

McKay, R .

SCIENCE, 1997, 276 (5309) :66-71

[64] Brain transplantation of genetically engineered human neural stem cells globally corrects brain lesions in the mucopolysaccharidosis type VII mouse [J].

Meng, XL ;

Shen, JS ;

Ohashi, T ;

Maeda, H ;

Kim, SU ;

Eto, Y .

JOURNAL OF NEUROSCIENCE RESEARCH, 2003, 74 (02) :266-277

[65] Transplanted bone marrow generates new neurons in human brains [J].

Mezey, É ;

Key, S ;

Vogelsang, G ;

Szalayova, I ;

Lange, GD ;

Crain, B .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (03) :1364-1369

[66] Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer [J].

Mingozzi, F ;

Liu, YL ;

Dobrzynski, E ;

Kaufhold, A ;

Liu, JH ;

Wang, YQ ;

Arruda, VR ;

High, KA ;

Herzog, RW .

JOURNAL OF CLINICAL INVESTIGATION, 2003, 111 (09) :1347-1356

[67] Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors [J].

Miyoshi, H ;

Smith, KA ;

Mosier, DE ;

Verma, IM ;

Torbett, BE .

SCIENCE, 1999, 283 (5402) :682-686

[68] Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy [J].

Mount, JD ;

Herzog, RW ;

Tillson, DM ;

Goodman, SA ;

Robinson, N ;

McCleland, ML ;

Bellinger, D ;

Nichols, TC ;

Arruda, VR ;

Lothrop, CD ;

High, KA .

BLOOD, 2002, 99 (08) :2670-2676

[69] In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector [J].

Naldini, L ;

Blomer, U ;

Gallay, P ;

Ory, D ;

Mulligan, R ;

Gage, FH ;

Verma, IM ;

Trono, D .

SCIENCE, 1996, 272 (5259) :263-267

[70]

NEUFELD EF, 1991, ANNU REV BIOCHEM, V60, P257, DOI 10.1146/annurev.biochem.60.1.257

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