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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Bibliografische gegevens
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2008
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Article
Online toegang: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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