Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Cell and gene therapies are moving towards correcting root causes of diseases. Let's take a look at future cell and gene therapy trends.

As in vivo cell therapies enter clinical trials, suppliers tackle the technical and logistical barriers to commercial ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...

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 ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...