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Ohio State University and colleagues from Beam Therapeutics have created lipid-based nanoparticles with messenger RNA. With their help, they were able to restore the production of the missing blood coagulation protein in mice with hemophilia and restore the process of removing cholesterol from the blood in animals with genetic pathology. The authors talked about their development on the pages of the journal Science Advances.

Two common variants in the KIF3A gene increase the risk of young children having a dysfunctional skin barrier and developing the skin condition atopic dermatitis. This, in turn, can allow environmental exposures to more easily cross the skin barrier and contribute to the development of food allergies and asthma as they grow up.

The U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This is the second FDA-approved targeted treatment for patients with this type of mutation. Approximately 8% of patients with DMD have a mutation that is amenable to exon 53 skipping.

A new approach to predicting which babies will develop type 1 diabetes moves a step closer to routine testing for newborns which could avoid life-threatening complications.

Genes that are thought to play a role in how the SARS-CoV-2 virus infects our cells have been found to be active in embryos as early as during the second week of pregnancy, say scientists at the University of Cambridge and the California Institute of Technology (Caltech).

NEW YORK – In a large genomics study of male infertility, researchers have discovered 36 candidate variants in 33 genes that were not previously connected to the condition, opening the door for new diagnostic methods and treatments.