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Gene therapy has been very helpful in treating autosomal recessive RP. In vivo gene editing can be a therapeutic option to treat adRP. Following electroporation of a CRISPR/Cas9 vector, we previously restored vision in neonatal adRP rats by selective ablation of the Rhodopsin S334ter transgene.
Source link: https://doi.org/10.3390/pharmaceutics14040824
Methods: The aim of this research was to determine whether in vivo somatic cell gene editing by adeno-associated virus in a mouse model could treat familial hypercholesterolemia caused by the Ldlr mutant in a mouse model. The AAV-CRISPR/Cas9 was created to correct a point mutation in the Ldlr gene in hepatocytes and was delivered subcutaneously to Ldlr E208X mice. After a high-fat diet regimen and that the Ldlr mutation was corrected in a subset of hepatocytes following AAV-CRISPR/Cas9 therapy, with LDLR protein expression partially restored. Conclusions: Our results show that in vivo AAV-CRISPR/Cas9–mediated Ldr gene mutations can partially restore LDLR expression and significantly reduce atherosclerosis phenotypes in Ldlr mutants, suggesting a potential therapeutic strategy for the therapy of patients with familial hypercholesterolemia.
Source link: https://doi.org/10.1161/circulationaha.119.042476
BACKGROUND The fourth most common cancer in women is breast papillomavirus infections. In the cell profiling assay, a significant number of infected cells in the sub-G1 phase was seen. In addition, the HPV-E6 gene mutation resulted in a significant rise in the level of P53 protein. Our results showed that AAV-mediated delivery of CRISPR/Cas9 can specifically target the HPV-E6 gene in HeLa cells, as well as its anti-proliferative effects, as well as local administration of this gene-editing device for HPV-related cervical cancers.
Source link: https://doi.org/10.1038/s41598-022-06025-w
The aged monkeys were more vulnerable to gene editing by showing faster PD progression, higher final total PD scores, and severer pathologic changes than their younger counterparts, indicating that both genetic and aging factors played important roles in PD development. This gene editing system can be used to produce a large number of genetically edited PD monkeys over a short time, thus providing a realistic transgenic monkey model for future PD research.
Source link: https://doi.org/10.1101/2020.09.19.305003
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