Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Albert M Maguire, Francesca Simonelli, Eric A Pierce, Edward N Pugh, Federico Mingozzi, Jeannette Bennicelli, Sandro Banfi, Kathleen A Marshall, Francesco Testa, Enrico M Surace, Settimio Rossi, Arkady Lyubarsky, Valder R Arruda, Barbara Konkle, Edwin Stone, Junwei Sun, Jonathan Jacobs, Lou Dell'Osso, Richard Hertle, Jian-xing Ma, T Michael Redmond, Xiaosong Zhu, Bernd Hauck, Olga Zelenaia, Kenneth S Shindler, Maureen G Maguire, J Fraser Wright, Nicholas J Volpe, Jennifer Wellman McDonnell, Alberto Auricchio, Katherine A High, Jean Bennett
The New England journal of medicine 2008;358(21):2240-8