共 23 条
[1]
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
[J].
Amoasii, Leonela
;
Hildyard, John C. W.
;
Li, Hui
;
Sanchez-Ortiz, Efrain
;
Mireault, Alex
;
Caballero, Daniel
;
Harron, Rachel
;
Stathopoulou, Thaleia-Rengina
;
Massey, Claire
;
Shelton, John M.
;
Bassel-Duby, Rhonda
;
Piercy, Richard J.
;
Olson, Eric N.
.
SCIENCE,
2018, 362 (6410)
:86-90

Amoasii, Leonela
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA
Exon Therapeut, 75 Kneeland St, Boston, MA 02111 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Hildyard, John C. W.
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Vet Coll, Dept Clin Sci & Serv, Comparat Neuromuscular Dis Lab, London NW1 0TU, England Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Li, Hui
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Sanchez-Ortiz, Efrain
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Mireault, Alex
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Caballero, Daniel
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Harron, Rachel
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Vet Coll, Dept Clin Sci & Serv, Comparat Neuromuscular Dis Lab, London NW1 0TU, England Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Stathopoulou, Thaleia-Rengina
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Vet Coll, Sect Anaesthesia & Analgesia, London NW1 0TU, England Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Massey, Claire
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Vet Coll, Dept Clin Sci & Serv, Comparat Neuromuscular Dis Lab, London NW1 0TU, England Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Shelton, John M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Internal Med, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Bassel-Duby, Rhonda
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Piercy, Richard J.
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Vet Coll, Dept Clin Sci & Serv, Comparat Neuromuscular Dis Lab, London NW1 0TU, England Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

Olson, Eric N.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Hamon Ctr Regenerat Sci & Med, Sen Paul D Wellstone Muscular Dystrophy Cooperat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA
[2]
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
[J].
Bennett, Jean
;
Wellman, Jennifer
;
Marshall, Kathleen A.
;
McCague, Sarah
;
Ashtari, Manzar
;
DiStefano-Pappas, Julie
;
Elci, Okan U.
;
Chung, Daniel C.
;
Sun, Junwei
;
Wright, J. Fraser
;
Cross, Dominique R.
;
Aravand, Puya
;
Cyckowski, Laura L.
;
Bennicelli, Jeannette L.
;
Mingozzi, Federico
;
Auricchio, Alberto
;
Pierce, Eric A.
;
Ruggiero, Jason
;
Leroy, Bart P.
;
Simonelli, Francesca
;
High, Katherine A.
;
Maguire, Albert M.
.
LANCET,
2016, 388 (10045)
:661-672

Bennett, Jean
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Wellman, Jennifer
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Spark Therapeut, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Marshall, Kathleen A.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

McCague, Sarah
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Ashtari, Manzar
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Dept Radiol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

DiStefano-Pappas, Julie
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Westat Biostat & Data Management Core, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Elci, Okan U.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Westat Biostat & Data Management Core, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Chung, Daniel C.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA
Spark Therapeut, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Sun, Junwei
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Wright, J. Fraser
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Spark Therapeut, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Cross, Dominique R.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Aravand, Puya
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Cyckowski, Laura L.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Dept Radiol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Bennicelli, Jeannette L.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Mingozzi, Federico
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Genethon, Immunol & Liver Gene Therapy, Evry, France Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Auricchio, Alberto
论文数: 0 引用数: 0
h-index: 0
机构:
Telethon Inst Genet & Med, Naples, Italy
Univ Naples Federico II, Med Genet, Dept Pediat, Naples, Italy Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Pierce, Eric A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA
Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Dept Ophthalmol, Philadelphia, PA 19104 USA
Harvard Med Sch, Ocular Genom Inst, Massachusetts Eye & Ear Infirm, Boston, MA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Ruggiero, Jason
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

论文数: 引用数:
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机构:

Simonelli, Francesca
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Naples 2, Eye Clin, Multidisciplinary Dept Med Surg & Dent Sci, Naples, Italy Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

High, Katherine A.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Spark Therapeut, Philadelphia, PA USA
Howard Hughes Med Inst, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA

Maguire, Albert M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA
Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA
[3]
T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS
[J].
BLAESE, RM
;
CULVER, KW
;
MILLER, AD
;
CARTER, CS
;
FLEISHER, T
;
CLERICI, M
;
SHEARER, G
;
CHANG, L
;
CHIANG, YW
;
TOLSTOSHEV, P
;
GREENBLATT, JJ
;
ROSENBERG, SA
;
KLEIN, H
;
BERGER, M
;
MULLEN, CA
;
RAMSEY, WJ
;
MUUL, L
;
MORGAN, RA
;
ANDERSON, WF
.
SCIENCE,
1995, 270 (5235)
:475-480

BLAESE, RM
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

CULVER, KW
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

MILLER, AD
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

CARTER, CS
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

FLEISHER, T
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

CLERICI, M
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

SHEARER, G
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

CHANG, L
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

CHIANG, YW
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

TOLSTOSHEV, P
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

GREENBLATT, JJ
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

ROSENBERG, SA
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

KLEIN, H
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

BERGER, M
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

MULLEN, CA
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

RAMSEY, WJ
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

MUUL, L
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

MORGAN, RA
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA

ANDERSON, WF
论文数: 0 引用数: 0
h-index: 0
机构: NCI, BETHESDA, MD 20892 USA
[4]
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice
[J].
Cantore, Alessio
;
Nair, Nisha
;
Della Valle, Patrizia
;
Di Matteo, Mario
;
Matrai, Janka
;
Sanvito, Francesca
;
Brombin, Chiara
;
Di Serio, Clelia
;
D'Angelo, Armando
;
Chuah, Marinee
;
Naldini, Luigi
;
VandenDriessche, Thierry
.
BLOOD,
2012, 120 (23)
:4517-4520

Cantore, Alessio
论文数: 0 引用数: 0
h-index: 0
机构:
Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Nair, Nisha
论文数: 0 引用数: 0
h-index: 0
机构:
Free Univ Brussels, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Della Valle, Patrizia
论文数: 0 引用数: 0
h-index: 0
机构:
Ist Sci San Raffaele, Coagulat Serv, I-20132 Milan, Italy
Ist Sci San Raffaele, Thrombosis Res Unit, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Di Matteo, Mario
论文数: 0 引用数: 0
h-index: 0
机构:
Free Univ Brussels, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium
Univ Louvain, Ctr Mol & Vasc Biol, Louvain, Belgium Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Matrai, Janka
论文数: 0 引用数: 0
h-index: 0
机构:
Free Univ Brussels, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium
Univ Louvain, Ctr Mol & Vasc Biol, Louvain, Belgium Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Sanvito, Francesca
论文数: 0 引用数: 0
h-index: 0
机构:
Ist Sci San Raffaele, Pathol Unit, Dept Oncol, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Brombin, Chiara
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Vita Salute San Raffaele, Univ Ctr Stat Biomed Sci, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Di Serio, Clelia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Vita Salute San Raffaele, Univ Ctr Stat Biomed Sci, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

D'Angelo, Armando
论文数: 0 引用数: 0
h-index: 0
机构:
Ist Sci San Raffaele, Coagulat Serv, I-20132 Milan, Italy
Ist Sci San Raffaele, Thrombosis Res Unit, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Chuah, Marinee
论文数: 0 引用数: 0
h-index: 0
机构:
Free Univ Brussels, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium
Univ Louvain, Ctr Mol & Vasc Biol, Louvain, Belgium Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

Naldini, Luigi
论文数: 0 引用数: 0
h-index: 0
机构:
Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy

VandenDriessche, Thierry
论文数: 0 引用数: 0
h-index: 0
机构:
Free Univ Brussels, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium
Univ Louvain, Ctr Mol & Vasc Biol, Louvain, Belgium Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, Italy
[5]
Gene Therapy for Hemoglobinopathies
[J].
Cavazzana, Marina
;
Mavilio, Fulvio
.
HUMAN GENE THERAPY,
2018, 29 (10)
:1106-1113

论文数: 引用数:
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Mavilio, Fulvio
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, IMAGINE Inst, Sorbonne Paris Cite, Paris, France
Univ Modena & Reggio Emilia, Dept Life Sci, Via Campi 287, I-41100 Modena, Italy Univ Paris 05, IMAGINE Inst, Sorbonne Paris Cite, Paris, France
[6]
Multiplex Genome Engineering Using CRISPR/Cas Systems
[J].
Cong, Le
;
Ran, F. Ann
;
Cox, David
;
Lin, Shuailiang
;
Barretto, Robert
;
Habib, Naomi
;
Hsu, Patrick D.
;
Wu, Xuebing
;
Jiang, Wenyan
;
Marraffini, Luciano A.
;
Zhang, Feng
.
SCIENCE,
2013, 339 (6121)
:819-823

Cong, Le
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA
MIT, Dept Biol Engn, Dept Brain & Cognit Sci, McGovern Inst Brain Res, Cambridge, MA 02139 USA
Harvard Univ, Sch Med, Program Biol & Biomed Sci, Boston, MA 02115 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Ran, F. Ann
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA
MIT, Dept Biol Engn, Dept Brain & Cognit Sci, McGovern Inst Brain Res, Cambridge, MA 02139 USA
Harvard Univ, Dept Mol & Cellular Biol, Cambridge, MA 02138 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

论文数: 引用数:
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机构:

Lin, Shuailiang
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA
MIT, Dept Biol Engn, Dept Brain & Cognit Sci, McGovern Inst Brain Res, Cambridge, MA 02139 USA
Tsinghua Univ, Sch Life Sci, Beijing 100084, Peoples R China Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Barretto, Robert
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Coll Phys & Surg, Dept Biochem & Mol Biophys, New York, NY 10032 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Habib, Naomi
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA
MIT, Dept Biol Engn, Dept Brain & Cognit Sci, McGovern Inst Brain Res, Cambridge, MA 02139 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Hsu, Patrick D.
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA
MIT, Dept Biol Engn, Dept Brain & Cognit Sci, McGovern Inst Brain Res, Cambridge, MA 02139 USA
Harvard Univ, Dept Mol & Cellular Biol, Cambridge, MA 02138 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Wu, Xuebing
论文数: 0 引用数: 0
h-index: 0
机构:
MIT, Computat & Syst Biol Grad Program, Cambridge, MA 02139 USA
MIT, Koch Inst Integrat Canc Res, Cambridge, MA 02139 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Jiang, Wenyan
论文数: 0 引用数: 0
h-index: 0
机构:
Rockefeller Univ, Bacteriol Lab, New York, NY 10065 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Marraffini, Luciano A.
论文数: 0 引用数: 0
h-index: 0
机构:
Rockefeller Univ, Bacteriol Lab, New York, NY 10065 USA Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA

Zhang, Feng
论文数: 0 引用数: 0
h-index: 0
机构:
Broad Inst MIT & Harvard, Cambridge Ctr 7, Cambridge, MA 02142 USA
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George, L. A.
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Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Sullivan, S. K.
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h-index: 0
机构:
Mississippi Ctr Adv Med, Dept Pediat, Madison, MS USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Giermasz, A.
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h-index: 0
机构:
Univ Calif Davis, Sch Med, Dept Med, Sacramento, CA 95817 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Rasko, J. E. J.
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h-index: 0
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Univ Sydney, Sydney Med Sch, Dept Med, Camperdown, NSW, Australia
Univ Sydney, Centenary Inst, Gene & Stem Cell Therapy Program, Camperdown, NSW, Australia
Royal Prince Alfred Hosp, Cell & Mol Therapies, Camperdown, NSW, Australia Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Samelson-Jones, B. J.
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Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Ducore, J.
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h-index: 0
机构:
Univ Calif Davis, Sch Med, Dept Pediat, Sacramento, CA 95817 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Cuker, A.
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h-index: 0
机构:
Univ Penn, Perelman Sch Med, Dept Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Sullivan, L. M.
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h-index: 0
机构:
Univ Mississippi, Med Sch, Dept Pathol, Jackson, MS USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Majumdar, S.
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h-index: 0
机构:
Univ Mississippi, Med Sch, Dept Pediat, Jackson, MS USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Teitel, J.
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h-index: 0
机构:
Univ Toronto, Fac Med, Dept Med, Toronto, ON, Canada
St Michaels Hosp, Toronto, ON, Canada Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

McGuinn, C. E.
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h-index: 0
机构:
Weill Cornell Med Coll, Dept Pediat, New York, NY USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Ragni, M. V.
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Univ Pittsburgh, Dept Med, Pittsburgh, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Luk, A. Y.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Hui, D.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Wright, J. F.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Chen, Y.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Liu, Y.
论文数: 0 引用数: 0
h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Wachtel, K.
论文数: 0 引用数: 0
h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Winters, 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, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Tiefenbacher, S.
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机构:
Lab Corp Amer Holdings, Colorado Coagulat, Englewood, CO USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Arruda, V. R.
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h-index: 0
机构:
Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
Univ Penn, Perelman Sch Med, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

van der Loo, J. C. M.
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h-index: 0
机构:
Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Zelenaia, O.
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h-index: 0
机构:
Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Takefman, D.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Carr, M. E.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Couto, L. B.
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h-index: 0
机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

Anguela, X. M.
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机构:
Spark Therapeut, Philadelphia, PA USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

High, K. A.
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Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France
Grp Hosp Univ Ouest, Ctr Invest Clin Integre Biotherapies, INSERM, Paris, France
Univ Paris 05, U768, INSERM, Paris, France
Hop Necker Enfants Malad, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

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Wang, Gary P.
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Univ Penn, Sch Med, Dept Microbiol, Philadelphia, PA 19104 USA Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Soulier, Jean
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h-index: 0
机构: Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Lim, Annick
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h-index: 0
机构:
Inst Pasteur, Dept Immunol, Unite Dev Lymphocytes, F-75724 Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Morillon, Estelle
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h-index: 0
机构:
Univ Paris 05, U768, INSERM, Paris, France
Hop Necker Enfants Malad, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

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机构:

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机构:

Delabesse, Eric
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Univ Hosp Purpan, Ctr Physiopathol Toulouse Purpan, Hematol Lab, Toulouse, France
Univ Hosp Purpan, Ctr Physiopathol Toulouse Purpan, U563, INSERM, Toulouse, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Beldjord, Kheira
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h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, Hematol Lab, Paris, France
Hop Necker Enfants Malad, AP HP, EMIU0210, INSERM, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Asnafi, Vahid
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, Hematol Lab, Paris, France
Hop Necker Enfants Malad, AP HP, EMIU0210, INSERM, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Macintyre, Elizabeth
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, Hematol Lab, Paris, France
Hop Necker Enfants Malad, AP HP, EMIU0210, INSERM, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Dal Cortivo, Liliane
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France
Grp Hosp Univ Ouest, Ctr Invest Clin Integre Biotherapies, INSERM, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Radford, Isabelle
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, EMIU0210, INSERM, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Brousse, Nicole
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, Dept Pathol, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Sigaux, Francois
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 07, IUH, Paris, France
Hop St Louis, AP HP, Hematol Lab, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Moshous, Despina
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, Dept Pediat Immunohematol, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Hauer, Julia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, U768, INSERM, Paris, France
Hop Necker Enfants Malad, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Borkhardt, Arndt
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Dusseldorf, Dept Pediat Hematol Oncol & Clin Immunol, Dusseldorf, Germany Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Belohradsky, Bernd H.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Childrens Hosp Munich, Dept Infect Dis & Immunol, Munich, Germany Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Wintergerst, Uwe
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Childrens Hosp Munich, Dept Infect Dis & Immunol, Munich, Germany Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Velez, Maria C.
论文数: 0 引用数: 0
h-index: 0
机构:
Louisiana State Univ, Hlth Sci Ctr, Dept Pediat, New Orleans, LA USA Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Leiva, Lily
论文数: 0 引用数: 0
h-index: 0
机构:
Louisiana State Univ, Hlth Sci Ctr, Dept Pediat, New Orleans, LA USA Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Sorensen, Ricardo
论文数: 0 引用数: 0
h-index: 0
机构:
Louisiana State Univ, Hlth Sci Ctr, Dept Pediat, New Orleans, LA USA Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Wulffraat, Nicolas
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Immunol Sect, Dept Pediat, Utrecht, Netherlands Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Blanche, Stephane
论文数: 0 引用数: 0
h-index: 0
机构:
Hop Necker Enfants Malad, AP HP, Dept Pediat Immunohematol, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Bushman, Frederic D.
论文数: 0 引用数: 0
h-index: 0
机构: Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Fischer, Alain
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, U768, INSERM, Paris, France
Hop Necker Enfants Malad, Paris, France
Hop Necker Enfants Malad, AP HP, Dept Pediat Immunohematol, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

Cavazzana-Calvo, Marina
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France
Grp Hosp Univ Ouest, Ctr Invest Clin Integre Biotherapies, INSERM, Paris, France
Univ Paris 05, U768, INSERM, Paris, France
Hop Necker Enfants Malad, Paris, France Univ Paris 05, Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France
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June, Carl H.
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h-index: 0
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Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA
Univ Penn, Parker Inst Canc Immunotherapy, Philadelphia, PA 19104 USA
Perlman Sch Med, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA

O'Connor, Roddy S.
论文数: 0 引用数: 0
h-index: 0
机构:
Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA
Univ Penn, Parker Inst Canc Immunotherapy, Philadelphia, PA 19104 USA Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA

Kawalekar, Omkar U.
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h-index: 0
机构:
Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA

Ghassemi, Saba
论文数: 0 引用数: 0
h-index: 0
机构:
Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA
Univ Penn, Parker Inst Canc Immunotherapy, Philadelphia, PA 19104 USA Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA

Milone, Michael C.
论文数: 0 引用数: 0
h-index: 0
机构:
Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA
Perlman Sch Med, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Perlman Sch Med, Ctr Cellular Immunotherapies, Philadelphia, PA 19104 USA