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Molecular Therapy. Nucleic Acids
|
August 17, 2018
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Kalyan Dulla, Monica Aguila, Amelia Lane, et al.
Investigative Ophthalmology & Visual Science
|
July 30, 2025
RHO-Associated Retinitis Pigmentosa: Genetics, Phenotype, Natural History, Functional Assays, and Animal Model - In Preparation for Clinical Trials
Malena Daich Varela, Juan Carlos Romo-Aguas, Rosellina Guarascio, et al.
Nature Genetics
|
October 7, 2008
EYS, encoding an ortholog of Drosophila spacemaker, is mutated in autosomal recessive retinitis pigmentosa
Mai M Abd El-Aziz, Isabel Barragan, Ciara A O'Driscoll, et al.
American Journal of Human Genetics
|
July 16, 2013
Mutations in ARL2BP, encoding ADP-ribosylation-factor-like 2 binding protein, cause autosomal-recessive retinitis pigmentosa
Alice E Davidson, Nele Schwarz, Lina Zelinger, et al.
Human Molecular Genetics
|
January 10, 2017
Rescue of mutant rhodopsin traffic by metformin-induced AMPK activation accelerates photoreceptor degeneration
Dimitra Athanasiou, Monica Aguila, Chikwado A Opefi, et al.
Human Mutation
|
August 30, 2014
Three different cone opsin gene array mutational mechanisms with genotype-phenotype correlation and functional investigation of cone opsin variants
Jessica C Gardner, Gerald Liew, Ying-Hua Quan, et al.
Ebiomedicine
|
September 15, 2024
Tissue-specific TCF4 triplet repeat instability revealed by optical genome mapping
Christina Zarouchlioti, Stephanie Efthymiou, Stefano Facchini, et al.
Plos One
|
March 14, 2012
Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy
Mei Hong Tan, Donna S Mackay, Jill Cowing, et al.
Plos Genetics
|
May 7, 2024
Deciphering novel TCF4-driven mechanisms underlying a common triplet repeat expansion-mediated disease
Nihar Bhattacharyya, Niuzheng Chai, Nathaniel J Hafford-Tear, et al.
American Journal of Human Genetics
|
February 1, 2025
Inter-chromosomal insertions at Xq27.1 associated with retinal dystrophy induce dysregulation of LINC00632 and CDR1as/ciRS-7
Jessica C Gardner, Katarina Jovanovic, Daniele Ottaviani, et al.
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Search research articles
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Showing results (131-140 of 161) with videos related to
Sort By:
Page
of 17
Molecular Therapy. Nucleic Acids
|
August 17, 2018
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Kalyan Dulla, Monica Aguila, Amelia Lane, et al.
Investigative Ophthalmology & Visual Science
|
July 30, 2025
RHO-Associated Retinitis Pigmentosa: Genetics, Phenotype, Natural History, Functional Assays, and Animal Model - In Preparation for Clinical Trials
Malena Daich Varela, Juan Carlos Romo-Aguas, Rosellina Guarascio, et al.
Nature Genetics
|
October 7, 2008
EYS, encoding an ortholog of Drosophila spacemaker, is mutated in autosomal recessive retinitis pigmentosa
Mai M Abd El-Aziz, Isabel Barragan, Ciara A O'Driscoll, et al.
American Journal of Human Genetics
|
July 16, 2013
Mutations in ARL2BP, encoding ADP-ribosylation-factor-like 2 binding protein, cause autosomal-recessive retinitis pigmentosa
Alice E Davidson, Nele Schwarz, Lina Zelinger, et al.
Human Molecular Genetics
|
January 10, 2017
Rescue of mutant rhodopsin traffic by metformin-induced AMPK activation accelerates photoreceptor degeneration
Dimitra Athanasiou, Monica Aguila, Chikwado A Opefi, et al.
Human Mutation
|
August 30, 2014
Three different cone opsin gene array mutational mechanisms with genotype-phenotype correlation and functional investigation of cone opsin variants
Jessica C Gardner, Gerald Liew, Ying-Hua Quan, et al.
Ebiomedicine
|
September 15, 2024
Tissue-specific TCF4 triplet repeat instability revealed by optical genome mapping
Christina Zarouchlioti, Stephanie Efthymiou, Stefano Facchini, et al.
Plos One
|
March 14, 2012
Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy
Mei Hong Tan, Donna S Mackay, Jill Cowing, et al.
Plos Genetics
|
May 7, 2024
Deciphering novel TCF4-driven mechanisms underlying a common triplet repeat expansion-mediated disease
Nihar Bhattacharyya, Niuzheng Chai, Nathaniel J Hafford-Tear, et al.
American Journal of Human Genetics
|
February 1, 2025
Inter-chromosomal insertions at Xq27.1 associated with retinal dystrophy induce dysregulation of LINC00632 and CDR1as/ciRS-7
Jessica C Gardner, Katarina Jovanovic, Daniele Ottaviani, et al.
Page
of 17