ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons

INTRODUCTION: Aniridia is a rare congenital vision-loss disease caused by heterozygous variants in the PAX6 gene. There is no vision-saving therapy, but one exciting approach is to use CRISPR/Cas9 to permanently correct the causal genomic variants. Preclinical studies to develop such a therapy in an...

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Detalles Bibliográficos
Autores principales: Adair, Bethany A., Korecki, Andrea J., Djaksigulova, Diana, Wagner, Pamela K., Chiu, Nina Y., Lam, Siu Ling, Lengyell, Tess C., Leavitt, Blair R., Simpson, Elizabeth M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2023
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10287867/
https://www.ncbi.nlm.nih.gov/pubmed/37210469
http://dx.doi.org/10.1007/s40123-023-00729-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10287867/
https://www.ncbi.nlm.nih.gov/pubmed/37210469
http://dx.doi.org/10.1007/s40123-023-00729-6

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