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Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease

Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent and severe monogenic disorder, SCD has been long considered a logical candidate for gene therapy. Significant progress has been made in moving toward this goal. These efforts have provided substantial insight...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Blood
Prif Awduron: Hoban, Megan D., Orkin, Stuart H., Bauer, Daniel E.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Hematology 2016
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4760089/
https://ncbi.nlm.nih.gov/pubmed/26758916
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2015-09-618587
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