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Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.

ABSTRACT: After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.

SUBMITTER: Wagemaker G 

PROVIDER: S-EPMC4179920 | biostudies-literature | 2014 Oct

REPOSITORIES: biostudies-literature

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Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.

Wagemaker Gerard G  

Human gene therapy 20140917 10

After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease. ...[more]

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