5 Result(s)
within
Protocol
Laura A. Jenny
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Protocol
Prime Editing for the Installation and Correction of Mutations Causing Inherited Retinal Disease: A Brief Methodology
Inherited retinal diseases ( ) encompass a large heterogeneous group of rare blinding disorders whose etiology originates from in the 280 genes identified to date. Clustered regularly interspaced short palin...
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Protocol
Metabolite Extraction from RPE Cells and Retinas Related to Retinitis Pigmentosa
The application of metabolomics in ophthalmology helps to identify new biomarkers and elucidate disease mechanisms in different eye diseases, as well as aiding in the development of potential treatment options...
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Protocol
Culture of Human Retinal Explants for Ex Vivo Assessment of AAV Gene Delivery
Due to the clinically established safety and efficacy profile of recombinant adeno-associated viral (rAAV) vectors, they are considered the “go to” vector for retinal gene therapy. Design of a rAAV-mediated ge...
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Protocol
CRISPR Manipulations in Stem Cell Lines
Insights into genome engineering in cells have allowed researchers to cultivate and modify cells as organoids that display structural and phenotypic features of human diseases or normal health status. The gene...
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Protocol
Generation of Human iPSC-Derived Retinal Organoids for Assessment of AAV-Mediated Gene Delivery
Human retinal organoids derived from induced pluripotent stem cells (iPSCs) serve as a promising preclinical model for testing the safety and efficacy of viral gene therapy. Retinal organoids recapitulate the ...
