共 69 条
Gene Therapy for Hemophilia
被引:68
作者:

Nienhuis, Arthur W.
论文数: 0 引用数: 0
h-index: 0
机构:
St Jude Childrens Res Hosp, Dept Hematol, Div Expt Hematol, MS 341, Memphis, TN 38105 USA St Jude Childrens Res Hosp, Dept Hematol, Div Expt Hematol, MS 341, Memphis, TN 38105 USA

Nathwani, Amit C.
论文数: 0 引用数: 0
h-index: 0
机构:
UCL, Canc Inst, Dept Hematol, 72 Huntley St, London WC1E 6BT, England St Jude Childrens Res Hosp, Dept Hematol, Div Expt Hematol, MS 341, Memphis, TN 38105 USA

Davidoff, Andrew M.
论文数: 0 引用数: 0
h-index: 0
机构:
St Jude Childrens Res Hosp, Dept Surg, Memphis, TN 38105 USA St Jude Childrens Res Hosp, Dept Hematol, Div Expt Hematol, MS 341, Memphis, TN 38105 USA
机构:
[1] St Jude Childrens Res Hosp, Dept Hematol, Div Expt Hematol, MS 341, Memphis, TN 38105 USA
[2] UCL, Canc Inst, Dept Hematol, 72 Huntley St, London WC1E 6BT, England
[3] St Jude Childrens Res Hosp, Dept Surg, Memphis, TN 38105 USA
关键词:
ADENOASSOCIATED VIRAL VECTORS;
FACTOR-IX EXPRESSION;
HIGHLY EFFICIENT TRANSDUCTION;
NONHUMAN PRIMATE LIVER;
OF-THE-ART;
IN-VIVO;
AAV VECTOR;
VIRUS VECTORS;
FACTOR-VIII;
HUMAN HEPATOCYTES;
D O I:
10.1016/j.ymthe.2017.03.033
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:
The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that can be life-threatening if untreated. Conventional therapy requires frequent intravenous infusions of the missing coagulation protein (factor VIII [ FVIII] for hemophilia A and factor IX [FIX] for hemophilia B). However, a lasting cure through gene therapy has long been sought. After a series of successes in small and large animal models, this goal has finally been achieved in humans by in vivo gene transfer to the liver using adeno-associated viral (AAV) vectors. In fact, multiple recent clinical trials have shown therapeutic, and in some cases curative, expression. At the same time, cellular immune responses against the virus have emerged as an obstacle in humans, potentially resulting in loss of expression. Transient immune suppression protocols have been developed to blunt these responses. Here, we provide an overview of the clinical development of AAV gene transfer for hemophilia, as well as an outlook on future directions.
引用
收藏
页码:1163 / 1167
页数:5
相关论文
共 69 条
[1]
Gene therapy for immune tolerance induction in hemophilia with inhibitors
[J].
Arruda, V. R.
;
Samelson-Jones, B. J.
.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS,
2016, 14 (06)
:1121-1134

Arruda, V. R.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
Univ Penn, Perelman Ctr Cell & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

Samelson-Jones, B. J.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[2]
Obstacles and future of gene therapy for hemophilia
[J].
Arruda, Valder R.
;
Samelson-Jones, Ben J.
.
EXPERT OPINION ON ORPHAN DRUGS,
2015, 3 (09)
:997-1010

Arruda, Valder R.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
Univ Penn, Ctr Cell & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

Samelson-Jones, Ben J.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[3]
The AAV Vector Toolkit: Poised at the Clinical Crossroads
[J].
Asokan, Aravind
;
Schaffer, David V.
;
Samulski, R. Jude
.
MOLECULAR THERAPY,
2012, 20 (04)
:699-708

Asokan, Aravind
论文数: 0 引用数: 0
h-index: 0
机构:
Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
Univ N Carolina, Dept Genet, Chapel Hill, NC 27599 USA Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA

Schaffer, David V.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Berkeley, Dept Chem Engn, Berkeley, CA 94720 USA
Univ Calif Berkeley, Helen Wills Neurosci Inst, Berkeley, CA 94720 USA Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA

Samulski, R. Jude
论文数: 0 引用数: 0
h-index: 0
机构:
Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
Univ N Carolina, Dept Pharmacol, Chapel Hill, NC 27599 USA Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
[4]
Promoter less gene targeting without nucleases ameliorates haemophilia B in mice
[J].
Barzel, A.
;
Paulk, N. K.
;
Shi, Y.
;
Huang, Y.
;
Chu, K.
;
Zhang, F.
;
Valdmanis, P. N.
;
Spector, L. P.
;
Porteus, M. H.
;
Gaensler, K. M.
;
Kay, M. A.
.
NATURE,
2015, 517 (7534)
:360-U476

Barzel, A.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Paulk, N. K.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Shi, Y.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif San Francisco, Dept Med, San Francisco, CA 94143 USA Dept Pediat, Stanford, CA 94305 USA

Huang, Y.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Chu, K.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Zhang, F.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Valdmanis, P. N.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Spector, L. P.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Porteus, M. H.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA

Gaensler, K. M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif San Francisco, Dept Med, San Francisco, CA 94143 USA Dept Pediat, Stanford, CA 94305 USA

Kay, M. A.
论文数: 0 引用数: 0
h-index: 0
机构:
Dept Pediat, Stanford, CA 94305 USA
Dept Genet, Stanford, CA 94305 USA Dept Pediat, Stanford, CA 94305 USA
[5]
Newand improved AAVenues: current status of hemophilia B gene therapy
[J].
Brimble, Mark A.
;
Reiss, Ulrike M.
;
Nathwani, Amit C.
;
Davidoff, Andrew M.
.
EXPERT OPINION ON BIOLOGICAL THERAPY,
2016, 16 (01)
:79-92

Brimble, Mark A.
论文数: 0 引用数: 0
h-index: 0
机构:
St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale, Memphis, TN 38105 USA
UCL, Inst Canc, Dept Haematol, London WC1E 6DD, England St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale, Memphis, TN 38105 USA

Reiss, Ulrike M.
论文数: 0 引用数: 0
h-index: 0
机构:
St Jude Childrens Res Hosp, Clin Haematol, 332 N Lauderdale St, Memphis, TN 38105 USA St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale, Memphis, TN 38105 USA

Nathwani, Amit C.
论文数: 0 引用数: 0
h-index: 0
机构:
UCL, Inst Canc, Dept Haematol, London WC1E 6DD, England
Royal Free NHS Fdn Trust, Katherine Dormandy Haemophilia & Thrombosis Unit, London NW3 2QG, England
NHS Blood & Transplant, Oak House Reeds Crescent, Watford WD24 4QN, England St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale, Memphis, TN 38105 USA

Davidoff, Andrew M.
论文数: 0 引用数: 0
h-index: 0
机构:
St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale, Memphis, TN 38105 USA St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale, Memphis, TN 38105 USA
[6]
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
[J].
Buchlis, George
;
Podsakoff, Gregory M.
;
Radu, Antonetta
;
Hawk, Sarah M.
;
Flake, Alan W.
;
Mingozzi, Federico
;
High, Katherine A.
.
BLOOD,
2012, 119 (13)
:3038-3041

Buchlis, George
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Sch Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Podsakoff, Gregory M.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Radu, Antonetta
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Dept Surg, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Hawk, Sarah M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Oklahoma, Hlth Sci Ctr, Oklahoma City, OK USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Flake, Alan W.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Dept Surg, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Mingozzi, Federico
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

High, Katherine A.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Sch Med, Philadelphia, PA 19104 USA
Univ Penn, Howard Hughes Med Inst, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
[7]
Manufacturing of recombinant adeno-associated viral vectors for clinical trials
[J].
Clement, Nathalie
;
Grieger, Joshua C.
.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT,
2016, 3
:16002

Clement, Nathalie
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Florida, Powell Gene Therapy Ctr, Dept Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Dept Pediat, Gainesville, FL USA

Grieger, Joshua C.
论文数: 0 引用数: 0
h-index: 0
机构:
Bamboo Therapeut Inc, Chapel Hill, NC 27517 USA Univ Florida, Powell Gene Therapy Ctr, Dept Pediat, Gainesville, FL USA
[8]
Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV
[J].
Darby, Sarah C.
;
Kan, Sau Wan
;
Spooner, Rosemary J.
;
Giangrande, Paul L. F.
;
Hill, Frank G. H.
;
Hay, Charles R. M.
;
Lee, Christine A.
;
Ludlam, Christopher A.
;
Williams, Michael
.
BLOOD,
2007, 110 (03)
:815-825

Darby, Sarah C.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Kan, Sau Wan
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Spooner, Rosemary J.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Giangrande, Paul L. F.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Hill, Frank G. H.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Hay, Charles R. M.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Lee, Christine A.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

Ludlam, Christopher A.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Manchester, Manchester Royal Infirm, Dept Haematol, Manchester M13 9WL, Lancs, England

论文数: 引用数:
h-index:
机构:
[9]
Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models
[J].
Davidoff, AM
;
Gray, JT
;
Ng, CYC
;
Zhang, YB
;
Zhou, JF
;
Spence, Y
;
Bakar, Y
;
Nathwani, AC
.
MOLECULAR THERAPY,
2005, 11 (06)
:875-888

Davidoff, AM
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Gray, JT
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Ng, CYC
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Zhang, YB
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Zhou, JF
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Spence, Y
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Bakar, Y
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England

Nathwani, AC
论文数: 0 引用数: 0
h-index: 0
机构: UCL, Dept Haematol, London WC1E 6HX, England
[10]
Early clinical data raise the bar for hemophilia gene therapies
[J].
Dolgin, Elie
.
NATURE BIOTECHNOLOGY,
2016, 34 (10)
:999-1001

Dolgin, Elie
论文数: 0 引用数: 0
h-index: 0