论文信息 - Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration - 字舞流文

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

Jean Bennett | Gui-Shuang Ying | Francesco Testa | Kathleen A. Marshall | Settimio Rossi | Francesca Simonelli | Sandro Banfi | G. Ying | J. Bennett | E. Surace | S. Banfi | J. F. Wright | E. Pierce | K. High | A. Maguire | A. Auricchio | F. Testa | F. Mingozzi | J. Bennicelli | S. Rossi | Xiaosong Zhu | K. Shindler | Carmela Acerra | Junwei Sun | J. McDonnell | F. Simonelli | T Michael Redmond | J Fraser Wright | Eric A Pierce | Kenneth S Shindler | Alberto Auricchio | Federico Mingozzi | Albert M Maguire | Jeannette L Bennicelli | Kathleen Marshall | Enrico M Surace | Junwei Sun | Xiaosong Zhu | Carmela Ziviello | Carmela Acerra | Jennifer Wellman McDonnell | Katherine A High | C. Ziviello | J. F. Wright | G. Ying

[1] Alexander Sumaroka,et al. Vision 1 year after gene therapy for Leber's congenital amaurosis. , 2009, The New England journal of medicine.

[2] A. J. Roman,et al. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. , 2009, Human gene therapy.

[3] J. Lupski,et al. Mutations in SPATA7 cause Leber congenital amaurosis and juvenile retinitis pigmentosa. , 2009, American journal of human genetics.

[4] W. Hauswirth,et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. , 2008, Human gene therapy.

[5] Edwin M Stone,et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics , 2008, Proceedings of the National Academy of Sciences.

[6] R. Roepman,et al. Leber congenital amaurosis: Genes, proteins and disease mechanisms , 2008, Progress in Retinal and Eye Research.

[7] Nick Tyler,et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. , 2008, The New England journal of medicine.

[8] Kathleen A. Marshall,et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. , 2008, The New England journal of medicine.

[9] J. F. Wright,et al. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

[10] A. Ciccodicola,et al. Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients. , 2007, Investigative ophthalmology & visual science.

[11] David Eidelberg,et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial , 2007, The Lancet.

[12] M. Ozelo,et al. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

[13] R. Hertle,et al. Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. , 2006, Investigative ophthalmology & visual science.

[14] J. Rasko,et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response , 2006, Nature Medicine.

[15] W. Hauswirth,et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[16] T. Aleman,et al. Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[17] Krzysztof Palczewski,et al. Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis. , 2004, Investigative ophthalmology & visual science.

[18] M. Marmor,et al. Standard for clinical electroretinography (2004 update) , 2004, Documenta Ophthalmologica.

[19] A. D. den Hollander,et al. Molecular genetics of Leber congenital amaurosis. , 2002, Human molecular genetics.

[20] Jean Bennett,et al. Gene therapy restores vision in a canine model of childhood blindness , 2001, Nature Genetics.

[21] D L Mayer,et al. Vision in Leber congenital amaurosis. , 1996, Archives of ophthalmology.

[22] A Kawasaki,et al. Variability of the relative afferent pupillary defect. , 1996, American journal of ophthalmology.

[23] S. Awaya,et al. Changes in nystagmus after simultaneous surgery for bilateral congenital cataracts. , 1993, Japanese journal of ophthalmology.

[24] G. Fishman,et al. Variability of visual field measurements in normal subjects and patients with retinitis pigmentosa. , 1984, Archives of ophthalmology.

[25] J. L. Smith,et al. Periodic alternating nystagmus clearing after vitrectomy. , 1982, Journal of clinical neuro-ophthalmology.

[26] P. Francois,et al. [Stargardt's disease and fundus flavimaculatus]. , 1975, Archives d'ophtalmologie et revue generale d'ophtalmologie.