检索结果 - Alexandra V. Garafalo
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Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives 由 Alexandra V. Garafalo, Artur V. Cideciyan, Elise Héon, Rebecca Sheplock, Alexander Pearson, Caberry W. Yu, Alexander Sumaroka, Gustavo D. Aguirre, Samuel G. Jacobson
出版 2019Revisão -
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Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report 由 Artur V. Cideciyan, Samuel G. Jacobson, Allen C. Ho, Alexandra V. Garafalo, Alejandro J. Román, Alexander Sumaroka, Arun K. Krishnan, Małgorzata Świder, Michael R. Schwartz, Aniz Girach
出版 2021Artigo -
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EYS Mutations Causing Autosomal Recessive Retinitis Pigmentosa: Changes of Retinal Structure and Function with Disease Progression 由 David B. McGuigan, Elise Héon, Artur V. Cideciyan, Rinki Ratnapriya, Monica Lu, Alexander Sumaroka, Alejandro J. Román, Vaishnavi Batmanabane, Alexandra V. Garafalo, Edwin M. Stone, Anand Swaroop, Samuel G. Jacobson
出版 2017Artigo -
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Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations 由 Samuel G. Jacobson, Artur V. Cideciyan, Allen C. Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Román, Alexander Sumaroka, Vivian Wu, Arun K. Krishnan, Rebecca Sheplock, Sanford L. Boye, Bee-Lin Cheang, Vanessa Davidson, Catherine R. O’Riordan, Alexander M. Dizhoor, Sanford L. Boye
出版 2021Artigo -
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Night vision restored in days after decades of congenital blindness 由 Samuel G. Jacobson, Artur V. Cideciyan, Allen C. Ho, Alejandro J. Román, Vivian Wu, Alexandra V. Garafalo, Alexander Sumaroka, Arun K. Krishnan, Małgorzata Świder, Abraham A. Mascio, Christine N. Kay, Dan Yoon, Kenji P. Fujita, Sanford L. Boye, Igor V. Peshenko, Alexander M. Dizhoor, Sanford L. Boye
出版 2022Artigo -
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Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial 由 Stephen R. Russell, Arlene V. Drack, Artur V. Cideciyan, Samuel G. Jacobson, Bart P. Leroy, Caroline Van Cauwenbergh, Allen C. Ho, Alina V. Dumitrescu, Ian C. Han, M. Carmen Marcos Martin, Wanda Pfeifer, Elliott H. Sohn, Jean Walshire, Alexandra V. Garafalo, Arun K. Krishnan, Christian A. Powers, Alexander Sumaroka, Alejandro J. Román, Eva Vanhonsebrouck, Eltanara A. Jones, Fanny Nerinckx, Julie De Zaeytijd, Rob W.J. Collin, Carel B. Hoyng, Peter Adamson, Michael E. Cheetham, Michael R. Schwartz, Wilhelmina den Hollander, Friedrich Asmus, Gerard Platenburg, David M. Rodman, Aniz Girach
出版 2022Artigo
相关主题
Medicine
Ophthalmology
Retinal
Biology
Gene
Clinical trial
Genetics
Internal medicine
Neuroscience
Adverse effect
Ciliopathy
GUCY2D
Genetic enhancement
Guanylate cyclase 2C
Pathology
Phenotype
Retina
Retinal degeneration
Retinitis pigmentosa
Visual acuity
Audiology
Biochemistry
Bioinformatics
Blinding
Blindness
Ciliopathies
Clinical endpoint
Cognitive psychology
Confounding
Cyclase