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

Learn more about whether BioMarin Pharmaceutical Inc. or CRISPR Therapeutics AG is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Genetic engineers can design and assemble sophisticated gene circuits to program cells with new functions, but important ...

Once a drug target is identified, it often takes decades until a chemical compound is developed that can hit the bullseye and ...

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

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

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

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This important study examines the potential role of ARHGAP36 transcriptional regulation by FOXC1 in controlling sonic hedgehog signaling in human neuroblastoma. While there are many solid findings ...

By tweaking the epigenetic state of a single gene, Arc, inside memory-storing neurons, researchers have altered memory ...