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.
Publication types
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Disease Models, Animal
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Genetic Therapy* / adverse effects
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Genetic Therapy* / methods
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Genetic Vectors / administration & dosage
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Genetic Vectors / adverse effects
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Genetic Vectors / genetics*
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Hematopoietic Stem Cell Transplantation
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Hematopoietic Stem Cells / metabolism*
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Humans
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Lentivirus / genetics*
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Metabolism, Inborn Errors / genetics*
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Metabolism, Inborn Errors / therapy*
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Mice
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Treatment Outcome