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

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.

doi:10.1016/j.omtm.2018.07.002

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

Journal Article · Sat Sep 01 00:00:00 EDT 2018 · Molecular Therapy. Methods & Clinical Development

DOI:https://doi.org/10.1016/j.omtm.2018.07.002· OSTI ID:1462278

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.

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Sponsoring Organization:: USDOE Office of Science (SC), Basic Energy Sciences (BES)

Grant/Contract Number:: Z01 HD008927; R01 GM090016; Z01 HD008768; AC02-06CH11357

OSTI ID:: 1462278

Journal Information:: Molecular Therapy. Methods & Clinical Development, Journal Name: Molecular Therapy. Methods & Clinical Development Vol. 10 Journal Issue: C; ISSN 2329-0501

Publisher:: ElsevierCopyright Statement

Country of Publication:: United States

Language:: English

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Cited by: 14 works

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References (46)

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