The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred.

Explore CRISPR technology explained, gene editing science, DNA modification, and genetic engineering ethics—discover how ...

A patient with transthyretin amyloidosis (ATTR) who received the investigational gene therapy nexiguran ziclumeran (nex-z) in ...

Recognized in the Biotechnology category of the 2025 Top Innovations contest, small Cas12l nucleases are versatile, ...

Technology opens the door to more accurate preclinical testing, which could enable the development of safer and more effective therapies.

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Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ...

Jarome and his team found that aging disrupts K63 polyubiquitination in two distinct areas of the brain. In the hippocampus, ...

Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists. Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in ...

The baby's disease prevented the liver from removing ammonia Half of babies with the disease die in their first week of life The baby has already shown signs of improvement The baby's disease ...

Emerging genetics research could help brands create mainstream wheat products that are safer for gluten-sensitive consumers – ...