Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

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 ...

The remarkable advancements in cell and gene therapy (CGT) have ushered in a new era of medical possibilities, with the recent FDA approval of the first CRISPR therapy for sickle cell gene editing ...

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, ...

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 ...

This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...