They successfully edited the gene in mice in vivo and their approach could also work for a range of heterozygous disease-causing SNPs.
Exome sequencing poorly covers some known disease genes, but identifies novel genes that that Harvard team thinks will help improve panel's diagnostic rate.
The partnership will specifically include STP601, Sirnaomics' preclinical treatment for ocular neovascularization diseases, in the Greater China and Southeast Asian markets.
In Science this week: genetic target for urothelial bladder cancer treatment, and more.
At the Conversation, the University of Oxford's Michael Macklay writes that learning genetic risk of disease is a personal decision.
Two dozen scientific organizations have endorsed the March for Science, according to ScienceInsider.
Researchers in Japan describe a chimpanzee with a chromosomal abnormality similar to human Down syndrome, Mashable reports.