Rare & Genetic Diseases

Not only does aging affect people differently, but it also affects their organs at varying rates too.

Improved chemistry may make RNA editing therapies more feasible in humans.

Age-related macular degeneration is the most common cause of vision loss in adults over age 50, but there are few treatments available. Researchers are now developing promising stem cell therapies to treat the disease.

Researchers are developing gene and cell therapies and prosthetics to help patients with blindness regain some vision. The first major prosthetic, the Argus II, was just discontinued. Where will the field go from here?

Engineered particles improve the efficiency of base editor delivery.

Creative solutions may enable gene therapy for more common diseases.

Recurrent high fevers in children are always worrying, but by studying patient cells, immunologist Lori Broderick hopes to reveal the underlying causes of these disorders.

LIVE March 28th, 2022 at 3:30 PM
Stem cell therapy—the holy grail of regenerative medicine—is finally entering the clinic.

From tuberculosis to drug-resistant bacterial infections, infectious diseases that plagued the globe long before COVID-19 still affect people today.

Recent advancements in mitochondrial genome editing technologies take scientists one step closer to developing viable treatments for mitochondrial diseases, which affect 1 in 4300 adults.

Researchers pioneered a new strategy for diagnosing puzzling genetic diseases, expanding scientists’ capability to study their causes and pathophysiology.

Philip Yeske was an organic chemist for more than 20 years, but when his daughter died from the rare mitochondrial disorder Leigh Syndrome, he turned to connecting researchers, patients, and law makers to speed the discovery of treatments.