An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

CORONA, Calif. (KABC) -- A teenage girl is alive thanks to an experimental gene therapy treatment created at UCLA. She was born with a rare genetic disorder, so any common infection could've killed ...

For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she could not make ...

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 ...

Cell and gene therapy are revolutionary approaches to treating diseases at the molecular level. They involve the manipulation ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in ...

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, ...

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...

According to DataM Intelligence, the global cell and gene therapy market reached a value of US$13.90 billion in 2024 and is ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. The New York pharma is ending global development and commercialization of Beqvez ...