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Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders

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 deficiencie...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile nagusia: Wagemaker, Gerard
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2014
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC4179920/
https://ncbi.nlm.nih.gov/pubmed/25184354
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2014.102
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