The Telegraph reports that UK regulators gave CRISPR Therapeutics the go-ahead for its beta thalassemia clinical trial.
CRISPR Therapeutics is moving toward clinical trials for beta thalassemia and sickle cell disease treatments, according to Wired.
The researchers plan to use the method in clinical research, designing noninvasive prenatal tests for single-gene disorders like beta thalassemia.
Using base editing, a Chinese research team has repaired a β-thalassemia-causing mutation in cloned embryos, Nature News reports.
In PNAS this week: work toward a CRISPR/Cas9-based approach to treat beta-thalassemia or sickle cell disease, bacteriophages uncovered in human stool, and more.
In Nature this week: Exome Aggregation Consortium analysis of some 60,000 exomes, and more.
Scientists introduced an indel to a promoter region to mimic a benign, naturally-occurring mutation that activated fetal hemoglobin production.
Though the edit was made in non-viable zygotes, the study wades into human germline engineering, a topic of great consternation for the CRISPR/Cas9 research community.
Alnylam Pharmaceuticals this week released new preclinical data from programs in beta-thalassemia and erythropoiesis — indications from which the company could select its newest pipeline candidate.
Scientists from China's Xiamen University and a startup company called Xiamen Zeesan Biotech have submitted the test to Chinese regulatory officials for approval as a widespread prenatal diagnostic and screening tool for β-thalassemia.
The US Food and Drug Administration has approved Alnylam's RNAi-based therapy Onpattro, according to Stat News.
Harvard Medical School's Seth Cassel and Cigall Kadoch argue in a Baltimore Sun op-ed that the recent TAILORx trial shows the potential of genomic-based medicine.
Researchers in the UK are working on using gene drives to control malaria-carrying mosquitoes, the Telegraph reports.
In PLOS this week: genetic architecture mediating gene expression, metabolomic patterns in multiple myeloma, and more.