Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease

Author:

Herstine Jessica A.ORCID,Chang Pi-Kai,Chornyy Sergiy,Stevenson Tamara J.,Sunshine Alex C.,Nokhrina Ksenia,Rediger Jessica,Wentz Julia,Vetter Tatyana A.,Scholl Erika,Holaway Caleb,Pyne Nettie K.,Bratasz Anna,Yeoh Stewart,Flanigan Kevin M.,Bonkowsky Joshua L.,Bradbury Allison M.ORCID

Funder

National Center for Advancing Translational Sciences

National Center for Research Resources

American Board of Psychiatry and Neurology Inc

Abigail Wexner Research Institute at Nationwide Children's Hospital

The Ohio State University Center for Clinical and Translational Science

Publisher

Elsevier BV

Reference41 articles.

1. Natural History of Vanishing White Matter;Hamilton;Ann. Neurol.,2018

2. A new leukoencephalopathy with vanishing white matter;van der Knaap;Neurology,1997

3. Vanishing white matter disease;van der Knaap;Lancet Neurol.,2006

4. [Clinical features of 54 cases of leukoencephalopathy with vanishing white matter disease in children];Zhou;Zhonghua Er Ke Za Zhi,2019

5. The effect of genotype on the natural history of eIF2B-related leukodystrophies;Fogli;Neurology,2004

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