Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and...
Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model
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United States: Elsevier Inc
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Language
English
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United States: Elsevier Inc
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Contents
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, using the mottled-brindled (mo-br) mouse model that cl...
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Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model
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TN_cdi_doaj_primary_oai_doaj_org_article_a4cc1ab740b44d4f9ac2ad5537b6c26f
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_a4cc1ab740b44d4f9ac2ad5537b6c26f
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ISSN
2329-0501
E-ISSN
2329-0501
DOI
10.1016/j.omtm.2018.07.002
