Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to appear soon after birth. To date, there haven’t been many reliable ...

CRISPR has the power to correct genetic mutations, but current delivery methods are either unsafe or inefficient, keeping the technology from reaching its full medical potential. With the power to ...

In the world of biopharmaceutical innovation, 2024 will be remembered as CRISPR’s breakout year. In the spring, five patients with sickle cell disease began treatment with Casgevy, the first ...

FOR 20 YEARS Tamani Harris lived a life of pain. She was born with sickle-cell disease. Her red blood cells, made flat and stiff by a mutant version of haemoglobin, struggled to move smoothly through ...

I was born in Washington, DC, in 1964, the oldest of three sisters, but the place that truly formed me as a scientist was Hilo, on the Big Island of Hawaii. Growing up there, far from the mainland ...

Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By ...

Scientists used CRISPR to disable the NRF2 gene, restoring chemotherapy sensitivity in lung cancer cells and slowing tumor growth. The technique worked even when only a fraction of tumor cells were ...