Collaborate Disseminate
Researchers at the University of California San Diego (UCSD) have demonstrated an intriguing new variation on the CRISPR gene-editing system, which involves fixing a mutation on one chromosome by copying a healthy version from the other. “Nicking” the … Continue reading New CRISPR tool corrects mutations by copying genes between chromosomes
As important as the CRISPR-Cas9 gene-editing tool has been, it has a relatively high error rate, which can introduce potentially harmful mutations. Researchers in Germany have now developed a more refined tool that reduces errors by nicking DNA instead… Continue reading New gene editing tool reduces errors by nicking DNA not cutting
Incredible new data presented recently at the European Hematology Association Congress has revealed an experimental CRISPR gene editing therapy is both safe and effective up to three years after treatment. The follow-up results come from one of the lon… Continue reading America’s first CRISPR trial is still nearly 100% effective 3 years on
In phase 1 trials, an experimental siRNA therapy has shown promise for reducing levels of a lipoprotein associated with a higher risk of premature heart disease. The treatment could help fight a form of the disease that can’t currently be treated throu… Continue reading siRNA therapy shows promise for reducing genetic heart disease risk
From diabetes to rare blood disease, CRISPR gene-editing technology is changing the way we tackle many threats to public health, and there are few bigger than those posed by vector-borne diseases carried by tiny critters. We’ve seen much exciting progr… Continue reading First ever gene-edited ticks offer new weapons against Lyme disease
Scientists at Boston Children’s Hospital have successfully used gene therapy to reverse a form of genetic hearing loss in mice. The team corrected a gene mutation that affects sensory hair cells in the inner ear, and the new jigsaw-like method could help improve gene therapy for other disorders.
Tags: Hearing, ear, gene therapy, Boston Children’s Hospital
Continue reading Jigsaw gene therapy reverses genetic hearing loss in mice
Researchers in China have developed a new three-pronged method to fight liver cancer that shows promise in tests in mice. The technique combines drugs and CRISPR-Cas9 gene editing into lipid nanoparticles, then activates them with ultrasound.Continue R… Continue reading CRISPR gene therapy, ultrasound and drugs team up against liver cancer
New research published in the journal Nature Communications has identified a potential treatment for chronic pain in an old experimental cancer drug. The research homed in on the drug by screening over a thousand different molecules in the search for o… Continue reading Experimental cancer drug offers clues to new kind of pain therapy
A long-term follow-up study tracking a group of children who received experimental gene therapy as part of an early-stage clinical trial is reporting the treatment is safe and effective up to 10 years later. The research offers rare insight into the lo… Continue reading One-off gene therapy for rare immune disease still effective 10 years later
One of the most revolutionary discoveries of the century, the CRISPR gene-editing system is built on a bacterial defense mechanism that allows scientists to make precise edits to DNA. Now scientists at MIT have discovered a new class of enzymes called … Continue reading Beyond CRISPR: New class of gene-editing enzymes discovered