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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 20, 2024
Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4
Nuria Suárez-Herrera, Iris B Riswick, Irene Vázquez-Domínguez, et al.
Progress in Retinal and Eye Research
|
August 17, 2025
Eyes shut homolog (EYS): Connecting molecule to disease
João Pedro Marques, Inês Santos Sousa, Daniela Patrício, et al.
Genes
|
February 2, 2018
Autosomal Recessive NRL Mutations in Patients with Enhanced S-Cone Syndrome
Karin W Littink, Patricia T Y Stappers, Frans C C Riemslag, et al.
Genes
|
March 10, 2018
Correction: Littink, K. W.; et al. Autosomal Recessive NRL Mutations in Patients with Enhanced S-Cone Syndrome. Genes 2018, 9, 68
Karin W Littink, Patricia T Y Stappers, Frans C C Riemslag, et al.
Cells
|
January 21, 2023
PDE6D Mediates Trafficking of Prenylated Proteins NIM1K and UBL3 to Primary Cilia
Siebren Faber, Stef J F Letteboer, Katrin Junger, et al.
Acta Neuropathologica Communications
|
February 11, 2025
Small molecule treatment alleviates photoreceptor cilia defects in LCA5-deficient human retinal organoids
Dimitra Athanasiou, Tess A V Afanasyeva, Niuzheng Chai, et al.
Cells
|
April 12, 2024
Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare <i>ABCA4</i> Variant in a Child with Early-Onset Stargardt Disease
Nuria Suárez-Herrera, Catherina H Z Li, Nico Leijsten, et al.
Genome Research
|
November 23, 2017
<i>ABCA4</i> midigenes reveal the full splice spectrum of all reported noncanonical splice site variants in Stargardt disease
Riccardo Sangermano, Mubeen Khan, Stéphanie S Cornelis, et al.
Progress in Retinal and Eye Research
|
July 8, 2021
Approaches for corneal endothelium regenerative medicine
Pere Català, Gilles Thuret, Heli Skottman, et al.
Progress in Retinal and Eye Research
|
March 30, 2018
Non-syndromic retinitis pigmentosa
Sanne K Verbakel, Ramon A C van Huet, Camiel J F Boon, et al.
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of 15
Search research articles
Search
Showing results (71-80 of 149) with videos related to
Sort By:
Page
of 15
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 20, 2024
Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4
Nuria Suárez-Herrera, Iris B Riswick, Irene Vázquez-Domínguez, et al.
Progress in Retinal and Eye Research
|
August 17, 2025
Eyes shut homolog (EYS): Connecting molecule to disease
João Pedro Marques, Inês Santos Sousa, Daniela Patrício, et al.
Genes
|
February 2, 2018
Autosomal Recessive NRL Mutations in Patients with Enhanced S-Cone Syndrome
Karin W Littink, Patricia T Y Stappers, Frans C C Riemslag, et al.
Genes
|
March 10, 2018
Correction: Littink, K. W.; et al. Autosomal Recessive NRL Mutations in Patients with Enhanced S-Cone Syndrome. Genes 2018, 9, 68
Karin W Littink, Patricia T Y Stappers, Frans C C Riemslag, et al.
Cells
|
January 21, 2023
PDE6D Mediates Trafficking of Prenylated Proteins NIM1K and UBL3 to Primary Cilia
Siebren Faber, Stef J F Letteboer, Katrin Junger, et al.
Acta Neuropathologica Communications
|
February 11, 2025
Small molecule treatment alleviates photoreceptor cilia defects in LCA5-deficient human retinal organoids
Dimitra Athanasiou, Tess A V Afanasyeva, Niuzheng Chai, et al.
Cells
|
April 12, 2024
Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare <i>ABCA4</i> Variant in a Child with Early-Onset Stargardt Disease
Nuria Suárez-Herrera, Catherina H Z Li, Nico Leijsten, et al.
Genome Research
|
November 23, 2017
<i>ABCA4</i> midigenes reveal the full splice spectrum of all reported noncanonical splice site variants in Stargardt disease
Riccardo Sangermano, Mubeen Khan, Stéphanie S Cornelis, et al.
Progress in Retinal and Eye Research
|
July 8, 2021
Approaches for corneal endothelium regenerative medicine
Pere Català, Gilles Thuret, Heli Skottman, et al.
Progress in Retinal and Eye Research
|
March 30, 2018
Non-syndromic retinitis pigmentosa
Sanne K Verbakel, Ramon A C van Huet, Camiel J F Boon, et al.
Page
of 15