gene therapy

In the race to deliver successful gene therapies, frontrunner AAVs come head to head with underdog lipid nanoparticles. 

A discovery that goes back to the first studies of translation has become the topic of biotech buzz.

An aqueduct connecting the brain to the ear may make gene therapy for hearing loss less invasive.

Gene therapy may be the first step toward curing a rare genetic epilepsy.

By delivering mRNA to the skin of mice in utero, researchers showed a proof-of-concept for shuttling gene therapies to skin cells before birth.

In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.

This year’s crop of winning products features many with a clinical focus and others that represent significant advances in sequencing, single-cell analysis, and more.

No deaths had previously been associated with the muscle-wasting treatment Zolgensma.

Mice rendered infertile through ovary cell–targeting mutations gave birth to seemingly normal offspring through natural mating after a virus-based gene therapy was injected into their ovaries.

CRISPR-mediated removal of HIV can create small, infectious DNA molecules.

A first-of-its-kind gene therapy dramatically reduced misfolded protein levels in some clinical trial participants for up to six months and reduced levels in all participants for up to a year.

RNA editing has been in DNA editing’s shadow for nearly a decade, but recent investments in the technology could bring it into the limelight.

The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.