"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.
The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...
Genetic diseases are notoriously challenging to treat, especially when each condition requires a tailored approach. With over 10,000 known genetic disorders, developing individual therapies for each ...
A closer examination of the APOL1 gene in Black patients with kidney disease can provide more accurate diagnoses than current ...
A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...
Horses are just as much a part of New Mexico’s story as green chile and balloons. One local startup is hoping their research ...
Mayo Clinic researchers have identified a rare mutation in the MET gene that can directly cause metabolic dysfunction-associated steatotic liver disease. The mutation disrupts the liver’s ability to ...
Donor candidates have to be aged 18 to 60, blood type 0, in good general health, and be willing to undergo screening.
During the Boston Globe’s Rare Disease Summit, Dr. Wendy Chung, a physician-scientist and geneticist who leads the Department of Pediatrics at Boston Children’s Hospital, said parents could soon see ...
A new study published in Neurology, led by Queen Mary University of London, has revealed that people of South Asian, African ...
The 19-year-old, who lives in Kelowna, had been diagnosed with chronic granulomatous disease around age five, compromising ...
A new federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...