Rare & Genetic Diseases

People metabolize drugs differently, changing the effectiveness and toxicity of drugs between individuals. Genetic testing to identify mutations in metabolic genes that may affect how someone processes a drug is an important step towards precision medicine that is becoming more common in the clinic.

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

By redirecting cells’ endogenous RNA editing machinery, scientists can correct disease-causing mutations, bringing a new kind of gene therapy to the clinic.

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.

Beyond the buzz, is AI just another research tool?

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 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.

LIVE July 21st, 2021 at 11:00 AM ET
This webinar will introduce DNAscope™, an innovative DNA ISH technology that employs a new probe design allowing for the precise targeting of small DNA stretches and convenient visualization under standard light microscopes.