Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

In one tiny town, more than a dozen people were diagnosed with the rare neurodegenerative disease ALS. Why? I n March 2009, ...

UCLA Health researchers say study findings uncover Huntington's disease mechanisms and pave new ways to develop treatments.

A federally funded research team led by Sheri Holmen, PhD, investigator at Huntsman Cancer Institute and professor in the ...

To fight this, doctors often use drugs called BRAF inhibitors, such as vemurafenib, which can stop the cancer’s growth—at ...

Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...

Preclinical data with the investigational gene therapy INS1202 show improvements in survival and motor performance in ALS.

For the first time, scientists say, they have evidence that using a biologic drug to remove sticky beta amyloid plaques from the brains<a class="excerpt-read-more" href=" More ...

Ovarian cancer (OC) is the fifth most common cause of cancer-related mortality worldwide and represents the leading cause of death for gynecological malignancies. Nevertheless, over the last years, ...

In a recent essay, scientists challenge the prevailing genetic-focused model of cancer, advocating for a shift towards more ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Understanding your tumor biology is key to finding the right treatment for pancreatic cancer. Identifying mutations, or changes in a [...] READ MORE ...