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Title
Gene therapy returns to centre stage
Authors
Keywords
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Journal
NATURE
Volume 526, Issue 7573, Pages 351-360
Publisher
Springer Nature
Online
2015-10-14
DOI
10.1038/nature15818
References
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Note: Only part of the references are listed.- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
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- (2015) Alan F. Wright NEW ENGLAND JOURNAL OF MEDICINE
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- (2015) Samuel G. Jacobson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Adoptive cell transfer as personalized immunotherapy for human cancer
- (2015) S. A. Rosenberg et al. SCIENCE
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- (2015) D. Baltimore et al. SCIENCE
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- (2015) T. N. Schumacher et al. SCIENCE
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- (2015) N. A. Rizvi et al. SCIENCE
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- (2015) Raffaella Greco et al. Frontiers in Pharmacology
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- (2014) A. Girard-Gagnepain et al. BLOOD
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- (2014) P. F. Robbins et al. CLINICAL CANCER RESEARCH
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- (2014) A. Barzel et al. NATURE
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- (2014) Troyen A Brennan et al. NATURE BIOTECHNOLOGY
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- (2010) Stefan Stein et al. NATURE MEDICINE
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- (2009) S Nayak et al. GENE THERAPY
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- (2009) Luigi D. Notarangelo et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
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- (2008) Naomi N. Hunder et al. NEW ENGLAND JOURNAL OF MEDICINE
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