Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders

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Wagemaker G.

HUMAN GENE THERAPY, vol.25, no.10, pp.862-865, 2014 (SCI-Expanded, Scopus)

• Publication Type: Article / Review
• Volume: 25 Issue: 10
• Publication Date: 2014
• Doi Number: 10.1089/hum.2014.102
• Journal Name: HUMAN GENE THERAPY
• Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
• Page Numbers: pp.862-865
• Open Archive Collection: AVESIS Open Access Collection
• Hacettepe University Affiliated: No

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.