A UCLA-led research team has developed a faster and more accurate way to determine where the many bacteria that live in, and on, humans come from. Broadly, the tool can deduce the origins of any microbiome, a localized and diverse community of microscopic organisms.
Scientists at Columbia have developed a gene-editing tool -- using jumping genes -- that inserts any DNA sequence into the genome without cutting, fixing a major shortcoming of existing CRISPR technology.
A China-US joint research team reported the generation of germline-transmittable cynomolgus macaques with Shank3 mutations, known to cause a form of autism.
The ability to hear depends on proteins to reach the outer membrane of sensory cells in the inner ear. But in certain types of hereditary hearing loss, mutations in the protein prevent it from reaching these membranes. Using a zebrafish model, researchers at Case Western Reserve University School of Medicine have found that an anti-malarial drug called artemisinin may help prevent hearing loss associated with this genetic disorder.
An international team of researchers led by the University of British Columbia has made a scientific advance they hope will lead to the development of preventative treatments for multiple sclerosis (MS).
Advances in preclinical research are now being translated into innovative clinical solutions for blindness, a review published in the 10th anniversary series of science Translational Medicine depicts.
Parkinson's disease (PD) is a progressive neurodegenerative disease affecting millions of people worldwide. Characterized by accumulation of the protein α-synuclein, there is currently no cure for PD. In a new study published this month in Scientific Reports, a research team led by Osaka University designed small fragments of DNA that bind to α-synuclein mRNA, preventing it from being translated into protein. This novel treatment effectively ameliorated disease symptoms in a mouse model of PD.
An internationally known embryologist and his son make the case for using gene-editing tools to prevent inherited disease, in an editorial published today in the journal Nature Medicine. The authors push back against recent calls for a moratorium on germline gene editing, following the revelation last year of the world's first gene-edited babies in China.
Researchers advancing gene-editing techniques to help patients with sickle cell disease discover an unexpected boost in fetal hemoglobin production, which mutes the effect of the disease.
Six months ago, a Chinese scientist announced that he had edited the genomes of two babies born last year. The germline edits with CRISPR-Cas9 supposedly changed the CCR5 gene to prevent HIV from invading immune cells. An analysis of records in the U.K. Biobank shows that having two copies of this mutation is associated with a 21% increase in mortality. UC Berkeley researchers caution against germline editing because of the unknown functions of many genes.