Month: July 2018

New studies raise warnings with CRISPR-Cas9, but there’s more to the story

We strive to stay at the forefront of any potentially groundbreaking therapy for our young patients, including gene therapy using the CRISPR-Cas9 platform. This gene-editing strategy allows scientists to cut DNA at specific locations in the genome and make targeted modifications with the ultimate hope of correcting disease-causing mutations. Since the original proof of concept papers were published in 2013, a flurry of research has been conducted in both academia and industry, and many groups are working hard to bring CRISPR-Cas9 to clinic. CRISPR-Cas9 therapies are especially exciting because they are not limited to one disease.  Additionally, CRISPR-Cas9 therapies...

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