Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
出版年份 2021 全文链接
标题
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
作者
关键词
-
出版物
Biomolecules
Volume 11, Issue 4, Pages 611
出版商
MDPI AG
发表日期
2021-04-21
DOI
10.3390/biom11040611
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- The Mechanism of Double-Strand DNA Break Repair by the Nonhomologous DNA End-Joining Pathway
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- Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience
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- Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype
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- A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells
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- Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
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- Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
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- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
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- SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors
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- Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease
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- Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB
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- AAV Vectors Avoid Inflammatory Signals Necessary to Render Transduced Hepatocyte Targets for Destructive T Cells
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- Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer
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- Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA
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- Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors
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