Genetics

Antisense oligonucleotides make sense in myotonic dystrophy

Antisense oligonucleotides – short segments of genetic material designed to target specific areas of a gene or chromosome – that activated an enzyme to "chew up" toxic RNA (ribonucleic acid) could point the way ...

Neuroscience

Lipid molecules may help get stroke therapies into the brain

To get therapies into the brain after a stroke, researchers are increasingly making use of the blood–brain barrier, which allows only certain molecules to pass from the blood into the brain. In a study published earlier ...

Medical research

A new treatment approach for cystic fibrosis

Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment ...

Oncology & Cancer

Cutting off liver cancer's nutrient supply chain

The World Health Organization projects that starting in 2030, over a million people will die each year from liver cancer. Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer, former postdoc Wai Kit Ma, and Dillon ...

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