Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia: Molecular Therapy - Nucleic Acids
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Sydney Sweeney Talks to Cosmo About Euphoria and Hollywood
Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer: Molecular Therapy
Caroline Wozniacki: The media needs to move on from Rory and me
Compartmentalized microfluidic device for in vitro co‐culture of retinal cells - Jahagirdar - 2022 - Biotechnology Journal - Wiley Online Library
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Inhibition of autophagy curtails visual loss in a model of autosomal dominant optic atrophy | Nature Communications
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration | Nature Communications
NMNAT1 mutations cause Leber congenital amaurosis | Nature Genetics
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Efficient correction of Duchenne muscular dystrophy mutations by SpCas9 and dual gRNAs: Molecular Therapy - Nucleic Acids
Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa - ScienceDirect
From The Archive: Alexa Chung On Her Lifelong Quest For The Perfect Jeans | British Vogue
Natural History of Cone Disease in the Murine Model of Leber Congenital Amaurosis Due to CEP290 Mutation: Determining the Timing and Expectation of Therapy | PLOS ONE
CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype | Gene Therapy
Alexa Chung: 'I don't know whether to be open and vulnerable' | Alexa Chung | The Guardian
Jason Leber - Senior Software Engineer - SONIFI Health | LinkedIn
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors: Molecular Therapy - Methods & Clinical Development
Frontiers | Exploiting hiPSCs in Leber's Hereditary Optic Neuropathy (LHON): Present Achievements and Future Perspectives
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Natural History of Cone Disease in the Murine Model of Leber Congenital Amaurosis Due to CEP290 Mutation: Determining the Timing and Expectation of Therapy | PLOS ONE
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis | Science Advances
Combination treatment of mannose and GalNAc conjugated small interfering RNA protects against lethal Marburg virus infection - ScienceDirect
