Reversing sickle cell disease with CRISPR-edited stem cells
August 1, 2019
By Neozene

Currently available gene therapies usually deliver a functional gene to replace a defective one. In a new approach developed at Fred Hutchinson Cancer Research Center, researchers instead introduced a mutation using the gene-editing system CRISPR-Cas9, and they’ve reported promising results from monkey studies aimed at boosting the production of fetal hemoglobin to treat blood disorders. Sickle cell disease and beta-thalassemia are caused by mutations in the beta-globin gene that cause misshapen red blood cells. Bluebird Bio’s Zynteglo, also known as LentiGlobin, adds functional copies of the gene to the patient’s

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Engineered bacteria prime solid tumors for immunotherapy
July 9, 2019
By Neozene

Arming the immune system so it can recognize and attack cancer is now a proven strategy for success in fighting some forms of the disease, notably blood cancers. But immunotherapy for solid tumors has proven to be elusive. Scientists at Columbia University hope to change that, using another emerging field of science: synthetic biology. A team at Columbia Engineering and the university’s Irving Medical Center are engineering bacteria that can deliver immunotherapy drugs straight to solid tumors. In mice, the strain of bacteria that the researchers made was able to

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Penn team finds new path to tumor death in mouse models of lymphoma and colon cancer
July 2, 2019
By Neozene

A cancer-causing gene called MYC has proven difficult to target with drugs, so oncology researchers have been looking for ways to combat it indirectly. Researchers at the University of Pennsylvania have found a new method for attacking MYC by targeting a protein that they believe is the oncogene’s Achilles’ heel. Blocking the protein, called ATF4, causes cancer cells to make too much of another type of protein family called 4E-BP. That causes the cancer cells so much stress that they die. The researchers believe that the discovery, published in the

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