Gene therapy for sickle cell disease: An update.
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ABSTRACT:
SUBMITTER: Demirci S
PROVIDER: S-EPMC6123269 | biostudies-literature | 2018 Jul
REPOSITORIES: biostudies-literature
Publications
Gene therapy for sickle cell disease: An update.
Demirci Selami S Uchida Naoya N Tisdale John F JF
Cytotherapy 20180530 7
Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates, but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies. Ex vivo gene therapy through globin gene addition has been investigated extensive ...[more]
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