Canavan Disease Latest Advances

Human iPSC-derived neural progenitor cells rescue motor function and brain pathology in symptomatic Canavan disease mice.

Journal: Stem cell reports

Published: July 21, 2025

Case 336: Canavan Disease.

Journal: Radiology

Published: April 29, 2025

Deep Intronic SVA_E Retrotransposition as a Novel Factor in Canavan Disease Pathogenesis.

Journal: Human gene therapy

Published: April 21, 2025

Neuroglia in leukodystrophies.

Journal: Handbook of clinical neurology

Published: March 27, 2025

Treatment of leukodystrophies: Advances and challenges.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: January 16, 2025

Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial.

Journal: Nature medicine

Published: December 31, 2024

Feasibility, Acceptability, and Reliability of Remote Motor Assessment in Children With Canavan Disease.

Journal: Pediatric neurology

Published: December 07, 2024

Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.

Journal: Human gene therapy

Published: December 04, 2024

Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.

Journal: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Published: June 11, 2024

Identification of potential pharmacological chaperones that selectively stabilize mutated Aspartoacylases in Canavan disease.

Journal: Biochimica et biophysica acta. Proteins and proteomics

Published: June 05, 2024

Myelin lesion in the aspartoacylase (Aspa) knockout rat, an animal model for Canavan disease.

Journal: Experimental animals

Published: March 27, 2024

Quantification of N-acetyl-l-aspartate in dried blood spots: A simple and fast LC-MS/MS neonatal screening method for the diagnosis of Canavan disease.

Journal: Molecular genetics and metabolism

Published: February 29, 2024

Canavan Disease Latest Advances

Find the Latest Research About Canavan Disease

Human iPSC-derived neural progenitor cells rescue motor function and brain pathology in symptomatic Canavan disease mice.

Human iPSC-derived neural progenitor cells rescue motor function and brain pathology in symptomatic Canavan disease mice.

Case 336: Canavan Disease.

Case 336: Canavan Disease.

Deep Intronic SVA_E Retrotransposition as a Novel Factor in Canavan Disease Pathogenesis.

Deep Intronic SVA_E Retrotransposition as a Novel Factor in Canavan Disease Pathogenesis.

Neuroglia in leukodystrophies.

Neuroglia in leukodystrophies.

Treatment of leukodystrophies: Advances and challenges.

Treatment of leukodystrophies: Advances and challenges.

Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial.

Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial.

Feasibility, Acceptability, and Reliability of Remote Motor Assessment in Children With Canavan Disease.

Feasibility, Acceptability, and Reliability of Remote Motor Assessment in Children With Canavan Disease.

Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.

Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.

Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.

Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.

Identification of potential pharmacological chaperones that selectively stabilize mutated Aspartoacylases in Canavan disease.

Identification of potential pharmacological chaperones that selectively stabilize mutated Aspartoacylases in Canavan disease.

Myelin lesion in the aspartoacylase (Aspa) knockout rat, an animal model for Canavan disease.

Myelin lesion in the aspartoacylase (Aspa) knockout rat, an animal model for Canavan disease.

Quantification of N-acetyl-l-aspartate in dried blood spots: A simple and fast LC-MS/MS neonatal screening method for the diagnosis of Canavan disease.

Quantification of N-acetyl-l-aspartate in dried blood spots: A simple and fast LC-MS/MS neonatal screening method for the diagnosis of Canavan disease.