CRISPR

Research news highlights a science breakthrough in CRISPR gene editing, and stem cell transplants provide curative potential for monogenic inflammatory bowel disease.

The landmark study paves the way for researchers to customize the technology and treat other individual patients with genetic diseases caused by ultra-rare “N-of-1” variants.

The Goldberg Lab is uncovering the mechanistic underpinnings of the disease, while studying a new form of genome editing as a potential fix.

Researchers are creating a platform to help fast-track personalized gene-editing therapies for patients with urea cycle disorders.

Using CRISPR/Cas9 gene editing in an early-stage clinical trial, researchers showed improved vision for a severe inherited form of blindness.

Evan Weber, PhD, and a research team from CHOP and Stanford Medicine discovered that the protein FOXO1 plays a key role in regulating CAR T cells’ longevity and effectiveness.

William Peranteau, MD, along with CHOP and Penn researchers are developing “one-and-done” new base-editing therapies for three genetic diseases of newborns.

After decades of limited progress in treating sickle cell disease, we have reached an historical moment with two new gene therapies.

This week’s research news features awards for furthering patient-centered outcomes research careers and for recognizing the importance of mentorship.

In this news roundup, we celebrate research tools new and old, from RNA sequencing to gene editing to the best tool of all: collaboration.