Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
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Author / Creator
Maguire, Maureen G , 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 , Wright, J. Fraser , Volpe, Nicholas J , McDonnell, Jennifer Wellman , Auricchio, Alberto , High, Katherine A and Bennett, Jean
Publisher
Boston, MA: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
Boston, MA: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Leber's congenital amaurosis (LCA) is a group of inherited disorders involving retinal degeneration with severe vision loss noted in early infancy. The condition is usually identified through behaviors, including abnormal roving-eye movements (nystagmus). The diagnosis is confirmed by both abnormal electroretinographic responses and pupillary light...
Alternative Titles
Full title
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Authors, Artists and Contributors
Author / Creator
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
Wright, J. Fraser
Volpe, Nicholas J
McDonnell, Jennifer Wellman
Auricchio, Alberto
High, Katherine A
Bennett, Jean
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_2829748
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_2829748
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa0802315
