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New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies
Inherited retinal dystrophies (IRDs) are a heterogeneous group of diseases that affect more than 2 million people worldwide. Gene therapy (GT) has emerged as an exciting treatment modality with the potential to provide long-term benefit to patients. Today, gene addition is the most straightforward G...
Autores principales: | Pulman, Juliette, Sahel, José-Alain, Dalkara, Deniz |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc., publishers
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9233507/ https://www.ncbi.nlm.nih.gov/pubmed/35506982 http://dx.doi.org/10.1089/crispr.2021.0141 |
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