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Open access

Research article

First published 2020-04-13

Practical management of lysosomal storage disorders (LSDs)

Pranoot Tanpaiboon

Translational Science of Rare Diseases

Open access

Review article

First published 2015

Lysosomal Storage Diseases-Regulating Neurodegeneration

Rob U. Onyenwoke, Jay E. Brenman

Neuroscience Insights

Retricted access

Research article

First published 2020-07

Development of an Antisense Oligonucleotide-Mediated Exon Skipping Therapeutic Strategy for Mucolipidosis II: Validation at RNA Level

Liliana Matos, Regina Vilela, Melissa Rocha [...]

Human Gene Therapy

Retricted access

Research article

First published 2005-7

Early Onset of Lysosomal Storage Disease in a Murine Model of Mucopolysaccharidosis Type VII: Undegraded Substrate Accumulates in Many Tissues in the Fetus and Very Young MPS VII Mouse

Carole Vogler, Beth Levy, Nancy Galvin [...]

Pediatric and Developmental Pathology

Open access

Review article

First published 2011-06

Identification and Characterization of Pharmacological Chaperones to Correct Enzyme Deficiencies in Lysosomal Storage Disorders

Kenneth J. Valenzano, Richie Khanna, Allan C. Powe [...]

ASSAY and Drug Development Technologies

Open access

Research article

First published 2025

Transplantation of Wild-Type Hematopoietic Stem and Progenitor Cells Improves Disease Phenotypes in a Mucopolysaccharidosis IIIC Mouse Model

Rafael A. Badell-Grau, Kasra Pakravesh, Kevin Eric Thai [...]

Cell Transplantation

Open access

Research article

First published 2017

Gene Therapy for Lysosomal Storage Disorders

Esteban Alberto Gonzalez, Guilherme Baldo

Journal of Inborn Errors of Metabolism and Screening

Retricted access

Review article

First published 2015-03

A Review of Gene Therapy in Canine and Feline Models of Lysosomal Storage Disorders

Allison M. Bradbury, Brittney L. Gurda, Margret L. Casal [...]

Human Gene Therapy Clinical Development

Open access

Research article

First published 2017

Newborn Screening for Lysosomal Storage Disorders in Belgium

Francois Eyskens, Sylvie Devos

Journal of Inborn Errors of Metabolism and Screening

Free access

Research article

First published 1984-7

A Lysosomal Storage Disorder in the BALB/c Mouse: Bone Marrow Transplantation

A. D. Boothe, H. Weintroub, P. G. Pentchev [...]

Veterinary Pathology

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