Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subretinal administration of adeno-associated vir...

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Veröffentlicht in:	Molecular therapy 2010-03, Vol.18 (3), p.643-650
Hauptverfasser:	Simonelli, Francesca, Maguire, Albert M, Testa, Francesco, Pierce, Eric A, Mingozzi, Federico, Bennicelli, Jeannette L, Rossi, Settimio, Marshall, Kathleen, Banfi, Sandro, Surace, Enrico M, Sun, Junwei, Redmond, T Michael, Zhu, Xiaosong, Shindler, Kenneth S, Ying, Gui-Shuang, Ziviello, Carmela, Acerra, Carmela, Wright, J Fraser, McDonnell, Jennifer Wellman, High, Katherine A, Bennett, Jean, Auricchio, Alberto
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Antibodies Carrier Proteins - genetics cis-trans-Isomerases Congenital diseases Dependovirus - genetics Disease Disease Progression Drug dosages Eye Proteins - genetics Follow-Up Studies Gene therapy Genetic Therapy - methods Genetic Vectors Humans Immunology Leber Congenital Amaurosis - genetics Leber Congenital Amaurosis - therapy Models, Genetic Mutation Ophthalmology Original Retina - metabolism Surgery Time Factors Transgenes Treatment Outcome Vision, Ocular Visual acuity
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