Genetic and functional correction of argininosuccinate lyase deficiency using CRISPR adenine base editors

Harnessing CRISPR to correct a metabolic inherited disease, we developed a therapeutically relevant method to precisely edit the Finnish Founder pathogenic variant responsible for argininosuccinate lyase deficiency. By lipid-nanoparticle-mediated delivery of the adenosine base editor RNA, we achieve efficient gene editing and metabolic normalization in patient fibroblasts.

Source: The American Journal of Human Genetics - April 4, 2024 Category: Genetics & Stem Cells Authors: Sami Jalil, Timo Keskinen, Juhana Juutila, Rocio Sartori Maldonado, Liliya Euro, Anu Suomalainen, Risto Lapatto, Emilia Kuuluvainen, Ville Hietakangas, Timo Otonkoski, Mervi E. Hyv önen, Kirmo Wartiovaara Tags: Article Source Type: research