Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model

Menkes disease is a lethal neurodegenerative disorder of copper metabolism caused by mutations in an evolutionarily conserved copper transporter, ATP7A. Based on our prior clinical and animal studies, we seek to develop a therapeutic approach suitable for application in affected human subjects, usin...

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Publicat a:Mol Ther Methods Clin Dev
Autors principals: Haddad, Marie Reine, Choi, Eun-Young, Zerfas, Patricia M., Yi, Ling, Martinelli, Diego, Sullivan, Patricia, Goldstein, David S., Centeno, Jose A., Brinster, Lauren R., Ralle, Martina, Kaler, Stephen G.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2018
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6080355/
https://ncbi.nlm.nih.gov/pubmed/30090842
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.07.002
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