rare diseases

Discover the most current cell therapy research and the challenges of translating them into the clinic.

May 12, 2022

Cell Therapy Advances: From Benchtop to Bedside

LIVE May 12th, 2022 at 2:30 PM
Discover the most current cell therapy research and the challenges of translating them into the clinic.

Mother measuring the temperature of her sleeping daughter

Mar 20, 2022By Stephanie DeMarco, PhD

Ultra-rare childhood fevers are no match for this scientist

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.

Discover how scientists develop successful stem cell therapies.

Mar 28, 2022

The Promise and Reality of Stem Cell Research

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

Mar 22, 2022

Using CRISPR to Develop Personalized Treatments

LIVE March 22nd, 2022 at 2:30 PM
Discover how researchers are correcting disease mutations with CRISPR genome editing.

ACOX1-driven neurodegeneration is one of the diseases that Bellen’s team has modeled in flies to find drug treatments. In a fly without ACOX1, the immune system attacks the fly’s own tissue.

Oct 04, 2021By Aparna Nathan

Genetic studies of rare undiagnosed diseases take flight with model organisms

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