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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Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2008
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Article
Länkar: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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