Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier-Reference-Cited by-同舟云学术

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Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier

Published:2015-02 Issue:2 Volume:114 Page:83-93
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Aronovich Elena L.,Hackett Perry B.

Funder

National Institutes of Health

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference149 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Pathogenic cascades in lysosomal disease—why so complex?;Walkley;J. Inherit. Metab. Dis.,2009

3. Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB;Ohmi;Proc. Natl. Acad. Sci. U. S. A.,2003

4. Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis;Jeyakumar;Brain,2003

5. Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity;Archer;J. Inherit. Metab. Dis.,2013

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1. Mapping brain networks in MPS I mice and their restoration following gene therapy;Scientific Reports;2023-08-05

2. Mucopolysaccharidosis: An overview and new treatment modalities;International Journal of Clinical Biochemistry and Research;2023-07-15

3. Liver directed adeno‐associated viral vectors to treat metabolic disease;Journal of Inherited Metabolic Disease;2023-06-05

4. Gene expression changes in Tay–Sachs disease begin early in fetal brain development;Journal of Inherited Metabolic Disease;2023-02-05

5. Clinical Development of Cell Therapies to Halt Lysosomal Storage Diseases: Results and Lessons Learned;Current Gene Therapy;2021-07-28

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