New findings show that some patients with a rare and aggressive form of leukodystrophy may benefit from receiving treatment with a class of targeted therapy drugs that could improve their neurological function.
Researchers report in Science Advances that the lipid-based nanoparticles they have engineered, carrying two sets of protein-making instructions, showed in animal studies that they have the potential to function as therapies for two genetic disorders.
UCLA scientists have developed a method for restoring oxygen-consumption activity to previously frozen mitochondria samples. By speeding up research, investigators hope to accelerate the diagnosis of people living with mitochondrial diseases and secondary disorders in which mitochondria play a key role, including diseases related to aging, metabolism and the heart.
New research is shedding light on the biological architecture that lets us hear – and on a genetic disorder that causes both deafness and blindness.