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 ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

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

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

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 ...

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 ...

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, ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

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

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 ...