Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8344120 | Molecular Genetics and Metabolism | 2012 | 7 Pages |
Abstract
⺠We treated a 3rd trimester Menkes disease fetus with in utero copper injections. ⺠Treatment increased fetal copper levels but did not improve copper transport. ⺠The patient developed signs and symptoms of the disease and died at 5.5 months. ⺠We suggest that postnatal viral gene therapy is needed for severe Menkes disease.
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Authors
Marie Reine Haddad, Charles J. Macri, Courtney S. Holmes, David S. Goldstein, Beryl E. Jacobson, Jose A. Centeno, Edwina J. Popek, Willam A. Gahl, Stephen G. Kaler,