This blog highlights research and development of new treatments, outcomes of global clinical trials and their regulations, with a special focus on ophthalmology and retinal diseases, including drugs and devices. The blog is meant for both clinical and pharmaceutical experts, as well as for patients / family members who are looking for information.
Scientists at Harvard University and the Broad Institute of MIT and Harvard have developed a new class of DNA base editor that can alter genomic structure to help repair the type of mutations that account for half of human disease-associated point mutations. These mutations are associated with disorders ranging from genetic blindness to sickle-cell anemia to metabolic disorders to cystic fibrosis.
