Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy-Reference-Cited by-同舟云学术

Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy

Published:2018-03 Issue:3 Volume:26 Page:874-889
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Marshall Michael S.,Issa Yazan,Jakubauskas Benas,Stoskute Monika,Elackattu Vince,Marshall Jeffrey N.,Bogue Wil,Nguyen Duc,Hauck Zane,Rue Emily,Karumuthil-Melethil Subha,Zaric Violeta,Bosland Maarten,van Breemen Richard B.,Givogri Maria I.,Gray Steven J.,Crocker Stephen J.,Bongarzone Ernesto R.

Funder

NRSA

NIH

Legacy of Angels Foundation and Research to Prevent Blindness

Legacy of Angels Foundation

Partners for Krabbe Disease Research

European Leukodystrophy Association

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference47 articles.

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2. Krabbe disease: a galactosylsphingosine (psychosine) lipidosis;Svennerholm;J. Lipid Res.,1980

3. Progressive accumulation of toxic metabolite in a genetic leukodystrophy;Igisu;Science,1984

4. The specificity of beta-galactosidase in the degradation of gangliosides;Suzuki;Adv. Exp. Med. Biol.,1980

5. A new familial, infantile form of diffuse brain sclerosis;Krabbe;Brain,1916

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