Gene Therapy in Hereditary Retinal Dystrophies: The Usefulness of Diagnostic Tools in Candidate Patient Selections

Purpose: Gene therapy actually seems to have promising results in the treatment of Leber Congenital Amaurosis and some different inherited retinal diseases (IRDs); the primary goal of this strategy is to change gene defects with a wild-type gene without defects in a DNA sequence to achieve partial r...

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Detalles Bibliográficos
Autores principales: Malvasi, Mariaelena, Casillo, Lorenzo, Avogaro, Filippo, Abbouda, Alessandro, Vingolo, Enzo Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10531171/
https://www.ncbi.nlm.nih.gov/pubmed/37762059
http://dx.doi.org/10.3390/ijms241813756

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10531171/
https://www.ncbi.nlm.nih.gov/pubmed/37762059
http://dx.doi.org/10.3390/ijms241813756

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