Canavan Disease

Canavan disease, a type of leukodystrophy, is an autosomal recessive neurodegenerative disorder, and is one of the most common degenerative cerebral diseases of infancy. Discover the latest research on Canavan disease here.

December 2, 2021

Selective screening for inborn errors of metabolism by tandem mass spectrometry at Sohag University Hospital, Egypt.

Archives de pédiatrie : organe officiel de la Sociéte française de pédiatrie
Rofaida M MagdyMontaser M Mohamed
November 2, 2021
Case Report

Alexander Disease.

Journal of the Belgian Society of Radiology
Laura HartogStephanie Vanden Bossche
September 3, 2021

High Throughput Screening Cascade To Identify Human Aspartate N-Acetyltransferase (ANAT) Inhibitors for Canavan Disease.

ACS Chemical Neuroscience
Ondřej NešutaBarbara S Slusher
August 27, 2021
Open Access

Canavan Disease: Clinical and Laboratory Profile from Southern Part of India.

Annals of Indian Academy of Neurology
Vykuntaraju K GowdaSanjay K Shivappa
May 20, 2021
Open Access

The natural history of Canavan disease: 23 new cases and comparison with patients from literature.

Orphanet Journal of Rare Diseases
Annette BleyFlorian S Eichler
May 10, 2021
Review
Open Access

Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair.

Frontiers in Cellular Neuroscience
Anoushka LotunGuangping Gao
April 30, 2021
Open Access

Mapping the degradation pathway of a disease-linked aspartoacylase variant.

PLoS Genetics
S. K. GersingRasmus Hartmann-Petersen
April 25, 2021
Review

Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.

Seminars in Pediatric Neurology
Allison M Bradbury, Margie A Ream
April 9, 2021

Engineering of a critical membrane-anchored enzyme for high solubility and catalytic activity.

Archives of Biochemistry and Biophysics
Muhammad S HussainRonald E Viola
March 30, 2021

Feline Spongy Encephalopathy With a Mutation in the ASPA Gene.

Veterinary Pathology
Yuta TakaichiKazuyuki Uchida
February 23, 2021
Open Access

Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease.

Molecular Therapy. Methods & Clinical Development
Jeremy S FrancisPaola Leone
December 15, 2020
Review
Open Access

Current Clinical Applications of In Vivo Gene Therapy with AAVs.

Molecular Therapy : the Journal of the American Society of Gene Therapy
Jerry R MendellJacques P Tremblay
December 12, 2020
Open Access

Cell-Based Therapy for Canavan Disease Using Human iPSC-Derived NPCs and OPCs.

Advanced Science
Lizhao FengYanhong Shi
November 4, 2020
Open Access

NGS-based expanded carrier screening for genetic disorders in North Indian population reveals unexpected results - a pilot study

BMC Medical Genetics
Kanika SinghIshwar Chander Verma
October 9, 2020
Preprint
Open Access

NGS based expanded pilot carrier screening study in North Indian population reveals unexpected results.

ResearchSquare
Kanika SinghIshwar Chander Verma

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