gene therapy

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.

Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.

In addition to isolating the first protein-coding gene from a eukaryote, Kedes furthered scientists’ understanding of actin genes and also laid the foundations for modern DNA databases such as GenBank.

Clinical trials were halted after the treatment’s vector that ferries in the healthy genetic sequence was identified in the genome of a patient’s cancer cells.

It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.

Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.