As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

This study reveals the potential of engineered phage-IL-12 therapy in melanoma, enhancing immune response and tumor ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Deepa Soundara Rajan, MD, and Jennifer L. Gannon, MD, discuss the current state of gene therapy for treating neurologic ...

The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, ...

This first-in-human trial is testing a highly unconventional method: delivering fat-derived stem cells from the patient ...

Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of ...

Find out if gene therapy has also find its way into skin diseases. With two approvals in the last 2 years, is it the start of ...

But, in some ways, he’s lucky. In June, the Food and Drug Administration expanded the use of Elevidys, a gene therapy, removing some of the previous restrictions to receive the treatment.