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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 2, 2017
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations
William A Beltran, Artur V Cideciyan, Shannon E Boye, et al.
American Journal of Human Genetics
|
May 5, 2009
Loss of the metalloprotease ADAM9 leads to cone-rod dystrophy in humans and retinal degeneration in mice
David A Parry, Carmel Toomes, Lina Bida, et al.
Archives of Ophthalmology (Chicago, Ill. : 1960)
|
September 14, 2011
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
Samuel G Jacobson, Artur V Cideciyan, Ramakrishna Ratnakaram, et al.
European Journal of Human Genetics : EJHG
|
January 20, 2005
CNGB3 mutations account for 50% of all cases with autosomal recessive achromatopsia
Susanne Kohl, Balazs Varsanyi, Gesine Abadin Antunes, et al.
Investigative Ophthalmology & Visual Science
|
November 15, 2012
Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease
Kari Branham, Mohammad Othman, Matthew Brumm, et al.
American Journal of Human Genetics
|
November 10, 2009
TRPM1 is mutated in patients with autosomal-recessive complete congenital stationary night blindness
Isabelle Audo, Susanne Kohl, Bart P Leroy, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
June 27, 2022
The landscape of submicroscopic structural variants at the <i>OPN1LW/OPN1MW</i> gene cluster on Xq28 underlying blue cone monochromacy
Bernd Wissinger, Britta Baumann, Elena Buena-Atienza, et al.
Nature Medicine
|
December 19, 2018
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Artur V Cideciyan, Samuel G Jacobson, Arlene V Drack, et al.
American Journal of Human Genetics
|
August 30, 2016
DNA Methylation Profiling of Uniparental Disomy Subjects Provides a Map of Parental Epigenetic Bias in the Human Genome
Ricky S Joshi, Paras Garg, Noah Zaitlen, et al.
Nature Medicine
|
April 5, 2022
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Stephen R Russell, Arlene V Drack, Artur V Cideciyan, et al.
Page
of 23
Search research articles
Search
Showing results (211-220 of 224) with videos related to
Sort By:
Page
of 23
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 2, 2017
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations
William A Beltran, Artur V Cideciyan, Shannon E Boye, et al.
American Journal of Human Genetics
|
May 5, 2009
Loss of the metalloprotease ADAM9 leads to cone-rod dystrophy in humans and retinal degeneration in mice
David A Parry, Carmel Toomes, Lina Bida, et al.
Archives of Ophthalmology (Chicago, Ill. : 1960)
|
September 14, 2011
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
Samuel G Jacobson, Artur V Cideciyan, Ramakrishna Ratnakaram, et al.
European Journal of Human Genetics : EJHG
|
January 20, 2005
CNGB3 mutations account for 50% of all cases with autosomal recessive achromatopsia
Susanne Kohl, Balazs Varsanyi, Gesine Abadin Antunes, et al.
Investigative Ophthalmology & Visual Science
|
November 15, 2012
Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease
Kari Branham, Mohammad Othman, Matthew Brumm, et al.
American Journal of Human Genetics
|
November 10, 2009
TRPM1 is mutated in patients with autosomal-recessive complete congenital stationary night blindness
Isabelle Audo, Susanne Kohl, Bart P Leroy, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
June 27, 2022
The landscape of submicroscopic structural variants at the <i>OPN1LW/OPN1MW</i> gene cluster on Xq28 underlying blue cone monochromacy
Bernd Wissinger, Britta Baumann, Elena Buena-Atienza, et al.
Nature Medicine
|
December 19, 2018
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Artur V Cideciyan, Samuel G Jacobson, Arlene V Drack, et al.
American Journal of Human Genetics
|
August 30, 2016
DNA Methylation Profiling of Uniparental Disomy Subjects Provides a Map of Parental Epigenetic Bias in the Human Genome
Ricky S Joshi, Paras Garg, Noah Zaitlen, et al.
Nature Medicine
|
April 5, 2022
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Stephen R Russell, Arlene V Drack, Artur V Cideciyan, et al.
Page
of 23