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 ...

Strategies Shaping Industry Growth. EINPresswire/ -- The Cell And Gene Therapy market is dominated by a mix of global biopharmaceutical leaders and specialized biotechnology innovators, with companies ...

Progress of in vivo gene therapy for use in malignancies, monogenic disorders, and degeneration disease. AAV, adeno-associated virus; TK, thymidine kinase; ADA-SCID, adenosine deaminase-deficient ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

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 ...

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 in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

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 ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...