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

ABSTRACT: 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 into the natural regulation of the globin genes and illuminated challenges for genetic manipulation of the hematopoietic system. The initial γ-retroviral vectors, next-generation lentiviral vectors, and novel genome engineering and gene regulation approaches each share the goal of preventing erythrocyte sickling. After years of preclinical studies, several clinical trials for SCD gene therapies are now open. This review focuses on progress made toward achieving gene therapy, the current state of the field, consideration of factors that may determine clinical success, and prospects for future development.

SUBMITTER: Hoban MD

PROVIDER: S-EPMC4760089 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature

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

Hoban Megan D MD Orkin Stuart H SH Bauer Daniel E DE

Blood 20160112 7

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 into the natural regulation of the globin genes and illuminated challenges for genetic manipulation of the hematopoietic system. The initial γ-retroviral vectors, next-generation lentiviral vectors, a ...[more]

PMID: 26758916

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