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Retinitis pigmentosa is a group of inherited, retinal degenerative disorders that are related to progressive rod photoreceptor loss and cone degeneration in early stages, resulting in vision loss or blindness. Gene therapy has been highly effective in treating autosomal recessive RP. Following electroporation of a CRISPR/Cas9 vector, we successfully restored vision in neonatal adRP rats by the selective ablation of the Rhodopsin S334ter transgene.
Source link: https://europepmc.org/article/MED/35456659
Gene therapy with adeno-associated viruses that do not nucleases has several advantages over traditional gene therapy, especially the possibility of permanent transgene expression. We investigated a number of small-molecule compounds and discovered that ribonucleotide reductase inhibitors significantly improved AAV-HR performance in mouse and human liver cell lines by threefold. In both fludarabine-treated and control mice, the overwhelming number of AAV-HR events occurred in non-proliferating hepatocytes, indicating that the induction of transient DNA repair signaling in non-dividing hepatocytes was responsible for increasing AAV-HR success in mice.
Source link: https://europepmc.org/article/MED/35393561
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