Sage Journals: Discover world-class research

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

Free access

Research article

First published 2021

Rare Variants Analysis of Lysosomal Related Genes in Early-Onset and Familial Parkinson’s Disease in a Chinese Cohort

Yong-Ping Chen, Xiao-Jing Gu, Wei Song [...]

Journal of Parkinson’s Disease

Retricted access

Research article

First published 2026-2

Intravenous Delivery of a Gene Therapy Vector that Expresses an Antitransferrin Receptor 1 Nanobody-I2S Fusion Protein Rescued Central Nervous System Lysosomal Burden in Mucopolysaccharidosis II Mice

Wanida Ruangsiriluk, Sarah Melissa P. Jacobo, Andrea Iskenderian [...]

Human Gene Therapy

Retricted access

Research article

First published 2025-4

Abnormal epigenetic modification of lysosome and lipid regulating genes in Alzheimer’s disease

Tingting Ruan, Yunxiang Ling, Can Wu [...]

Journal of Alzheimer’s Disease

Retricted access

Research article

First published 1991-09

Organization of the Gene Encoding Human Lysosomal β-Galactosidase

Hans Morreau, Erik Bonten, Xiao Yan Zhou [...]

DNA and Cell Biology

Retricted access

Research article

First published 2012-08

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

Barry J. Byrne, Darin J. Falk, Nathalie Clément [...]

Human Gene Therapy

Retricted access

Research article

First published 1999-07

Correction of Enzymatic and Lysosomal Storage Defects in Fabry Mice by Adenovirus-Mediated Gene Transfer

Robin J. Ziegler, Nelson S. Yew, Chester Li [...]

Human Gene Therapy

Retricted access

Research article

First published 1995-06

Correction of Deficient Enzyme Activity in a Lysosomal Storage Disease,Aspartylglucosaminuria,by Enzyme Replacement and Retroviral Gene Transfer

N. Enomaa, O. Danos, L. Peltonen [...]

Human Gene Therapy

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

Retricted access

Research article

First published 2025-2

Neuroprotective effects of chitinase-1 and calcitonin gene-related peptide on Alzheimer's disease by promoting lysosomal function

Wenkai Yang, Weihua Yu, Yang Lv

Journal of Alzheimer’s Disease

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