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Human Gene Therapy
|
December 7, 2011
Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept
Astra Dinculescu, Jackie Estreicher, Juan C Zenteno, et al.
Human Mutation
|
September 24, 2019
Deep-intronic variants in CNGB3 cause achromatopsia by pseudoexon activation
Nicole Weisschuh, Marc Sturm, Britta Baumann, et al.
American Journal of Human Genetics
|
January 14, 2003
Evaluation of complex inheritance involving the most common Bardet-Biedl syndrome locus (BBS1)
Kirk Mykytyn, Darryl Y Nishimura, Charles C Searby, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
Samuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.
Human Molecular Genetics
|
November 5, 2005
Retinal degeneration associated with RDH12 mutations results from decreased 11-cis retinal synthesis due to disruption of the visual cycle
Debra A Thompson, Andreas R Janecke, Jessica Lange, et al.
Human Gene Therapy
|
March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012
Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.
Plos One
|
January 15, 2016
Mutation Detection in Patients with Retinal Dystrophies Using Targeted Next Generation Sequencing
Nicole Weisschuh, Anja K Mayer, Tim M Strom, et al.
American Journal of Human Genetics
|
December 19, 2012
Whole-exome sequencing identifies LRIT3 mutations as a cause of autosomal-recessive complete congenital stationary night blindness
Christina Zeitz, Samuel G Jacobson, Christian P Hamel, et al.
Investigative Ophthalmology & Visual Science
|
December 23, 2021
Comparative Natural History of Visual Function From Patients With Biallelic Variants in BBS1 and BBS10
Monika K Grudzinska Pechhacker, Samuel G Jacobson, Arlene V Drack, et al.
The Journal of Clinical Investigation
|
March 27, 2012
Combining Cep290 and Mkks ciliopathy alleles in mice rescues sensory defects and restores ciliogenesis
Rivka A Rachel, Helen L May-Simera, Shobi Veleri, et al.
Page
of 23
Search research articles
Search
Showing results (201-210 of 224) with videos related to
Sort By:
Page
of 23
Human Gene Therapy
|
December 7, 2011
Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept
Astra Dinculescu, Jackie Estreicher, Juan C Zenteno, et al.
Human Mutation
|
September 24, 2019
Deep-intronic variants in CNGB3 cause achromatopsia by pseudoexon activation
Nicole Weisschuh, Marc Sturm, Britta Baumann, et al.
American Journal of Human Genetics
|
January 14, 2003
Evaluation of complex inheritance involving the most common Bardet-Biedl syndrome locus (BBS1)
Kirk Mykytyn, Darryl Y Nishimura, Charles C Searby, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
Samuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.
Human Molecular Genetics
|
November 5, 2005
Retinal degeneration associated with RDH12 mutations results from decreased 11-cis retinal synthesis due to disruption of the visual cycle
Debra A Thompson, Andreas R Janecke, Jessica Lange, et al.
Human Gene Therapy
|
March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012
Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.
Plos One
|
January 15, 2016
Mutation Detection in Patients with Retinal Dystrophies Using Targeted Next Generation Sequencing
Nicole Weisschuh, Anja K Mayer, Tim M Strom, et al.
American Journal of Human Genetics
|
December 19, 2012
Whole-exome sequencing identifies LRIT3 mutations as a cause of autosomal-recessive complete congenital stationary night blindness
Christina Zeitz, Samuel G Jacobson, Christian P Hamel, et al.
Investigative Ophthalmology & Visual Science
|
December 23, 2021
Comparative Natural History of Visual Function From Patients With Biallelic Variants in BBS1 and BBS10
Monika K Grudzinska Pechhacker, Samuel G Jacobson, Arlene V Drack, et al.
The Journal of Clinical Investigation
|
March 27, 2012
Combining Cep290 and Mkks ciliopathy alleles in mice rescues sensory defects and restores ciliogenesis
Rivka A Rachel, Helen L May-Simera, Shobi Veleri, et al.
Page
of 23