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Researchers have developed a new way to make virus-like particles more effective at delivering gene-editing tools into human cells by changing the cells that produce them.

Scientists from Purdue and Columbia universities describe an RNA-guided molecular machine that activates gene expression by recruiting the cell’s transcription machinery, offering a gentler alternative to standard gene-cutting tools.

UW–Madison researchers identify genes that block CRISPR success, offering a roadmap for safer gene therapy—particularly for inherited eye diseases.

Using AI protein modeling and a new screening system, scientists enhance a small DNA-editing enzyme to achieve higher efficiency with less unwanted damage and toxicity.

"NANITE" strategy amplifies genome editing in vivo by turning transfected cells into temporary factories that deliver Cas9 to their neighbors.

Researchers create a precise way to change DNA using artificial intelligence and gene tools, improving safety for modeling diseases and creating better therapies.

New research results suggest the possibility of finding ways to turn on new neuron production in old or injured brains.

MIT engineers develop a compact system to regulate gene expression for safer, effective treatments of genetic disorders.

The trial activates three genes to partially reprogram aged cells in an effort to regenerate damaged optic nerve neurons in glaucoma patients.

... but only works for patients with otoferln deficiency and may cost up to $250,000