Sage Journals: Discover world-class research

Open access

Research article

First published 2019

Global Brain Transcriptome Analysis of a Tpp1 Neuronal Ceroid Lipofuscinoses Mouse Model

Miriam S. Domowicz, Wen-Ching Chan, Patricia Claudio-Vázquez [...]

ASN Neuro

Open access

Research article

First published 2018

An Adapted Clinical Measurement Tool for the Key Symptoms of CLN2 Disease

Kathleen W. Wyrwich, Angela Schulz, Miriam Nickel [...]

Journal of Inborn Errors of Metabolism and Screening

Retricted access

Case report

First published 2025-1

Clinical,Pathological,and Molecular Findings in a Mexican Patient With Neuronal Ceroid Lipofuscinosis Type 2: Support for Pathogenicity of the c.1226 G>T Variant and for Presence of Cherry-Red Spot in This Disease

Celso Tomás Corcuera-Delgado, Alfonso Gilberto Ramírez-Ristori, Estela Pérez-Muñoz [...]

Pediatric and Developmental Pathology

Retricted access

Research article

First published 2023-09

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

Bishnu P. De, Jonathan B. Rosenberg, Nithya Selvan [...]

Human Gene Therapy

Retricted access

Research article

First published 2025-01

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease

Dolan Sondhi, Stephen M. Kaminsky, Jonathan B. Rosenberg [...]

Human Gene Therapy

Retricted access

Research article

First published 2004-11

Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis

Human Gene Therapy

Retricted access

Research article

First published 2005-12

Safety of Direct Administration of AAV2 CU hCLN2,a Candidate Treatment for the Central Nervous System Manifestations of Late Infantile Neuronal Ceroid Lipofuscinosis,to the Brain of Rats and Nonhuman Primates

Neil R. Hackett, D. Eugene Redmond, Dolan Sondhi [...]

Human Gene Therapy

Search results

Global Brain Transcriptome Analysis of a Tpp1 Neuronal Ceroid Lipofuscinoses Mouse Model

An Adapted Clinical Measurement Tool for the Key Symptoms of CLN2 Disease

Clinical,Pathological,and Molecular Findings in a Mexican Patient With Neuronal Ceroid Lipofuscinosis Type 2: Support for Pathogenicity of the c.1226 G>T Variant and for Presence of Cherry-Red Spot in This Disease

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease

Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis

Safety of Direct Administration of AAV2 CU hCLN2,a Candidate Treatment for the Central Nervous System Manifestations of Late Infantile Neuronal Ceroid Lipofuscinosis,to the Brain of Rats and Nonhuman Primates