Safety and Efficacy of Gene Transfer for Leber’s Congenital Amaurosis

Leber’s congenital amaurosis (LCA) is a group of inherited blinding diseases with onset during childhood. One form of the disease, LCA2, is caused by mutations in the retinal pigment epithelium–specific 65-kDa protein gene (RPE65). We investigated the safety of subretinal delivery of a recombinant a...

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Detalhes bibliográficos
Main Authors: Maguire, Albert M., Simonelli, Francesca, Pierce, Eric A., Pugh, Edward N., Mingozzi, Federico, Bennicelli, Jeannette, Banfi, Sandro, Marshall, Kathleen A., Testa, Francesco, Surace, Enrico M., Rossi, Settimio, Lyubarsky, Arkady, Arruda, Valder R., Konkle, Barbara, Stone, Edwin, Sun, Junwei, Jacobs, Jonathan, Dell’Osso, Lou, Hertle, Richard, Ma, Jian-xing, Redmond, T. Michael, Zhu, Xiaosong, Hauck, Bernd, Zelenaia, Olga, Shindler, Kenneth S., Maguire, Maureen G., Wright, J. Fraser, Volpe, Nicholas J., McDonnell, Jennifer Wellman, Auricchio, Alberto, High, Katherine A., Bennett, Jean
Formato: Artigo
Idioma:Inglês
Publicado em: 2008
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2829748/
https://ncbi.nlm.nih.gov/pubmed/18441370
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa0802315
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