Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

The Director of the San Raffaele-Telethon Institute for Gene Therapy Luigi Naldini reflects on what a recent Nature study reveals and about the future of in vivo gene transfer “Our study provides an ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. Subscribe ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

Researchers developed a gorilla adenovirus platform with inherent tumor selectivity, lung tropism, and large-gene capacity.

Three Philadelphia scientists won a $3 million Breakthrough Prize for developing the first FDA-approved gene therapy for a genetic disease, prize sponsors announced Saturday. Their work created a ...

DELRAY BEACH, FL / ACCESS Newswire / August 12, 2025 / Adolore BioTherapeutics, Inc., (“Company” or “Adolore”) announced that Roy Clifford Levitt, MD, Clinical Professor at the University of Miami, ...

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing (NGS) methods in nonclinical studies to assess the safety risks associated ...