title: 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.
authors.affiliation: Erasmus University Rotterdam, 3005 LA Rotterdam, The Netherlands .
authors.forename: Gerard
authors.initials: G
authors.lastname: Wagemaker
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage / Q000009:adverse effects / Q000235:genetics*; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism*; D006801:Humans; D016086:Lentivirus / Q000235:genetics*; D008661:Metabolism, Inborn Errors / Q000235:genetics* / Q000628:therapy*; D051379:Mice; D016896:Treatment Outcome