News

A new, alternative treatment for patients with wet age-related macular degeneration may be surgical removal of the abnormal blood vessels followed by the transplantation of stem cell-derived retinal cells.

A vast search of natural diversity has led scientists at MIT’s McGovern Institute and the Broad Institute of MIT and Harvard to uncover ancient systems with the potential to expand the genome editing toolbox.

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition.

A method developed by a research group at Tampere University seeks specific molecules that can target desired tissues through the bloodstream and penetrate tissues by passing through cells.

Stanford scientists discovered that sugar chains on brain cells, called the glycocalyx, degrade with age, weakening the blood-brain barrier. This allows harmful molecules to infiltrate the brain, fueling inflammation and cognitive decline.

MD Anderson researchers developed a novel antibody-toxin conjugate (ATC) that trains the immune system to fight cancer. Unlike traditional ADCs, this ATC targets CD47, marking tumors for immune destruction while delivering a bacterial toxin.

Following a more than two-year-long hold, Entrada Therapeutics can get its Duchenne muscular dystrophy candidate back on track. The company announced on Monday that the US FDA had lifted its clinical hold on ENTR-601-44.

A bone marrow transplant process co-developed by investigators at the Johns Hopkins Kimmel Cancer Center is safe and curative for adults with sickle cell disease, according to results of a trial.

Researchers have engineered two mycobacterial strains with built-in kill switches that trigger self-destruction after immune activation. These strains enhance the safety of high-dose intravenous BCG vaccines.

Researchers at Purdue University have developed NuFold, a machine learning tool that predicts 3D RNA structures from sequences. Dubbed the RNA equivalent of AlphaFold, NuFold bridges the gap in experimental RNA data.