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Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia | Synapse

April 18, 2018New England Journal of Medicine667 citationsOpen Access

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Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

ATAlexis A. Thompson MWMark C. Walters JKJanet L. Kwiatkowski

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Abstract

Donor availability and transplantation-related risks limit the broad use of allogeneic

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Thompson et al. (Wed,) studied this question.

synapsesocial.com/papers/69dcbfc225b1b6cb3335995e https://doi.org/https://doi.org/10.1056/nejmoa1705342

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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector1996 · 4,928 citations
2Pattern of complications and burden of disease in patients affected by beta thalassemia major2017 · 50 citations
3Elevated zinc protoporphyrin associated with thalassemia trait and hemoglobin E1996 · 45 citations
4A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency2014 · 411 citations
5Soluble form of transferrin receptor as a biomarker of overall morbidity in patients with non-transfusion-dependent thalassaemia: a cross-sectional study.2016 · 19 citations