Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.
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ABSTRACT:
SUBMITTER: Wagemaker G
PROVIDER: S-EPMC4179920 | biostudies-literature | 2014 Oct
REPOSITORIES: biostudies-literature
Publications
Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.
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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