research News

SHANGHAI and CLINTON, N.J. — HuidaGene Therapeutics today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys. Unlike the in-vivo use of Cas9 system with limited genome-editing efficiency, high occurrence...
Human fibrinogen concentrate (HFC) has favorable pharmacokinetics for adult, adolescent, and pediatric patients with congenital fibrinogen deficiency (CFD), according to the results of open-label studies presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting. The prospective studies FORMA-01, FORMA-02, and FORMA-04 looked at efficacy, safety, and dosing of...
LA JOLLA, CA—People who suffer from Peutz-Jeghers syndrome, a rare inherited cancer syndrome, develop gastrointestinal polyps and are predisposed to colon cancer and other tumor types. Carefully tracing the cellular chain-of-command that links nutrient intake to cell growth (and which is interrupted in Peutz-Jeghers syndrome), allowed researchers at the Salk...
Researchers at the University of Illinois at Chicago College of Medicine have discovered how the mutated huntingtin gene acts on the nervous system to create the devastation of Huntington’s disease. The report of their findings is available in Nature Neuroscience online. The researchers were able to show that the mutated...
HyVIS, the European project coordinated by the IIT-Istituto Italiano di Tecnologia (Italian Institute of Technology), is about to start. By combining nanotechnology and optics, it will develop bionic synapses for retinal prostheses, designed to restore sight in people suffering from diseases such as retinitis pigmentosa and age-related macular degeneration (AMD)....
INDIANAPOLIS — Clinical research led by Indiana University School of Medicine investigators and their collaborators in Uganda has revealed that hydroxyurea significantly reduces infections in children with sickle cell anemia. Their latest findings enhance strong evidence of hydroxyurea’s effectiveness and could ultimately reduce death in children in Africa, the continent...