Genetically modifying individual cells in animals

One proven method for tracking down the genetic causes of diseases is to knock out a single gene in animals and study the consequences this has for the organism. The problem is that for many diseases, the pathology is determined ...

Study shows promise of gene therapy for alcohol use disorder

A form of gene therapy currently used to treat Parkinson's disease may dramatically reduce alcohol use among chronic heavy drinkers, researchers at Oregon Health & Science University and institutions across the country have ...

Research team first to develop 3D structure of twinkle protein

Researchers from the National Institutes of Health have developed a three-dimensional structure that allows them to see how and where disease mutations on the twinkle protein can lead to mitochondrial diseases. The protein ...

A banner year for pharma

As 2018 draws to a close, the pharmaceutical industry is celebrating a prosperous year of new investments and therapeutic breakthroughs. These successes were driven by cutting-edge science and progress in finally translating ...

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