Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment

Barry J. Byrne; Darin J. Falk; Nathalie Clément; Cathryn S. Mah

doi:10.1089/hum.2012.140

Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment

Human Gene Therapy ◽

10.1089/hum.2012.140 ◽

2012 ◽

Vol 23 (8) ◽

pp. 808-815 ◽

Cited By ~ 39

Author(s):

Barry J. Byrne ◽

Darin J. Falk ◽

Nathalie Clément ◽

Cathryn S. Mah

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Next Generation ◽

Lysosomal Storage

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1035. Efficacy of Gene Therapy for a Prototypical Lysosomal Storage Disease (GSD-II) Is Critically Dependent upon Vector Dose, Transgene Promoter, and the Tissues Targeted for Vector Transduction

Molecular Therapy ◽

10.1016/s1525-0016(16)43865-7 ◽

2002 ◽

Vol 5 (5) ◽

pp. S336-S337

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage ◽

Vector Transduction ◽

Gsd Ii

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Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease

Molecular Therapy ◽

10.1038/mt.2012.100 ◽

2012 ◽

Vol 20 (7) ◽

pp. 1393-1399 ◽

Cited By ~ 31

Author(s):

Yong Hong Chen ◽

Kristin Claflin ◽

James C Geoghegan ◽

Beverly L Davidson

Keyword(s):

Gene Therapy ◽

Sialic Acid ◽

Mouse Model ◽

Acid Deposition ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage

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Efficacy of Gene Therapy for a Prototypical Lysosomal Storage Disease (GSD-II) Is Critically Dependent on Vector Dose, Transgene Promoter, and the Tissues Targeted for Vector Transduction

Molecular Therapy ◽

10.1006/mthe.2002.0563 ◽

2002 ◽

Vol 5 (4) ◽

pp. 436-446 ◽

Cited By ~ 48

Author(s):

Enyu Ding ◽

Huimin Hu ◽

Bradley L. Hodges ◽

Felicia Migone ◽

Delila Serra ◽

...

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage ◽

Vector Transduction ◽

Gsd Ii

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Brain-directed gene therapy for lysosomal storage disease: Going well beyond the blood- brain barrier

Proceedings of the National Academy of Sciences ◽

10.1073/pnas.102175599 ◽

2002 ◽

Vol 99 (9) ◽

pp. 5760-5762 ◽

Cited By ~ 37

Author(s):

W. S. Sly ◽

C. Vogler

Keyword(s):

Gene Therapy ◽

Blood Brain Barrier ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Brain Barrier ◽

Lysosomal Storage ◽

Blood Brain

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362. Gene Therapy Overcomes the Need for Multiple Infusions of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

Molecular Therapy ◽

10.1016/s1525-0016(16)35375-8 ◽

2014 ◽

Vol 22 ◽

pp. S138

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage ◽

Enzyme Replacement

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Gene therapy for lysosomal storage disease: A no-brainer?

Nature Medicine ◽

10.1038/nm0797-719 ◽

1997 ◽

Vol 3 (7) ◽

pp. 719-720 ◽

Cited By ~ 8

Author(s):

William S. Sly ◽

Carole Vogler

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage

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1040. Development of a Novel Xenotransplant Model (NOD/SCID/MPSVII) To Determine the Efficacy of Human Hematopoietic Progenitor Cell-Directed Gene Therapy for a Lysosomal Storage Disease

Molecular Therapy ◽

10.1016/s1525-0016(16)43870-0 ◽

2002 ◽

Vol 5 (5) ◽

pp. S338

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Disease ◽

Progenitor Cell ◽

Storage Disease ◽

Hematopoietic Progenitor Cell ◽

Lysosomal Storage ◽

Hematopoietic Progenitor

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Gene therapy/cell therapy for lysosomal storage disease

Journal of Inherited Metabolic Disease ◽

10.1023/a:1005692215317 ◽

2000 ◽

Vol 23 (3) ◽

pp. 293-298 ◽

Cited By ~ 6

Author(s):

Y. Eto ◽

T. Ohashi

Keyword(s):

Gene Therapy ◽

Cell Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage

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Gene therapy for lysosomal storage disease?

Inpharma Weekly ◽

10.2165/00128413-199710990-00019 ◽

1997 ◽

Vol &NA; (1099) ◽

pp. 11

Author(s):

&NA;

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Lysosomal Storage

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

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2014.09.011 ◽

2015 ◽

Vol 114 (2) ◽

pp. 83-93 ◽

Cited By ~ 32

Author(s):

Elena L. Aronovich ◽

Perry B. Hackett

Keyword(s):

Gene Therapy ◽

Blood Brain Barrier ◽

Lysosomal Storage Disease ◽

Storage Disease ◽

Disease Gene ◽

Brain Barrier ◽

Lysosomal Storage ◽

Blood Brain

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