Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates-Reference-Cited by-同舟云学术

Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates

Published:2023-09 Issue:9 Volume:31 Page:2767-2782
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Powers Samantha,Likhite Shibi,Gadalla Kamal K.,Miranda Carlos J.,Huffenberger Amy J.,Dennys Cassandra,Foust Kevin D.,Morales Pablo,Pierson Christopher R.,Rinaldi Federica,Perry Stephanie,Bolon Brad,Wein Nicolas,Cobb Stuart,Kaspar Brian K.,Meyer Kathrin C.^ORCID

Funder

Rett Syndrome Research Trust

Publisher

Elsevier BV

Subject

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

Reference60 articles.

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4. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2;Amir;Nat. Genet.,1999

5. Neonatal encephalopathy in two boys in families with recurrent Rett syndrome;Schanen;J. Child Neurol.,1998

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