Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities-Reference-Cited by-同舟云学术

Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities

Published:2022-06 Issue: Volume:25 Page:278-296
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Khoja Suhail,Lambert Jenna,Nitzahn Matthew,Eliav Adam,Zhang YuChen,Tamboline Mikayla,Le Colleen T.,Nasser Eram,Li Yunfeng,Patel Puja,Zhuravka Irina,Lueptow Lindsay M.,Tkachyova Ilona,Xu Shili,Nissim Itzhak,Schulze Andreas,Lipshutz Gerald S.

Funder

National Institutes of Health

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

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5. Mild guanidinoacetate increase under partial guanidinoacetate methyltransferase deficiency strongly affects brain cell development;Hanna-El-Daher;Neurobiol. Dis.,2015

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