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Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of Mngie
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by thymidine phosphorylase (TP) deficiency resulting in systemic accumulation of thymidine (d-Thd) and deoxyuridine ...
Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Immune and Lysosomal Enzyme Deficiencies
Rare diseases affect millions of people worldwide. Many of those are inherited disorders resulting in chronic disability and requiring cost-intensive care. Hematopoietic stem cell gene therapy has been developed over more ...
Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders
(Mary Ann Liebert, Inc, 2014)
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 ...