Rare & Genetic Diseases

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.

Researchers are using CRISPR gene editing to restore function of the mutant protein that causes Duchenne muscular dystrophy, a genetic disease that causes muscle degeneration.

An engineer turned biology researcher co-founded a bio-pharma start-up with the goal of using CRISPR to reverse the mutation causing the degenerative muscle disorder Duchenne muscular dystrophy.

LIVE October 25th, 2021 at 1:00 PM ET
Delivering CRISPR cargo in vivo to treat disease-causing DNA mutations

Researchers use fruit flies to figure out how genetic mutations disrupt biology and how drugs might reverse these effects.

CRISPR gene editing directly and indirectly drives advances in cardiovascular disease therapeutic development.

A native Hawaiian, anthropologist, and genome scientist, Keolu Fox investigates how mutations found in indigenous populations contribute to human health and emphasizes the importance of considering who benefits from research involving indigenous genomes.

LIVE August 26th, 2021 at 11:00 AM ET

In this webinar, experts will discuss new approaches to identify small therapeutic candidates targeting diabetes and Alzheimer’s disease.

LIVE July 28th, 2021 at 2:30 PM ET
In this webinar, experts will discuss cytosine and adenosine base-editors to introduce point mutations in DNA strands without creating a breakpoint, and opportunities and considerations for using them.