Team discovers new drug combo to induce high rates of human beta cell regeneration

Researchers at the Icahn School of Medicine at Mount Sinai have discovered a novel combination of two classes of drugs that, together, cause the highest rate of proliferation ever observed in adult human beta cells—the cells in the pancreas that produce insulin—without harming most other cells in the body. The result is an important step toward a diabetes treatment that restores the body’s ability to produce insulin.
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Designer probiotic treatment for cancer immunotherapy

Researchers at Columbia Engineering have engineered probiotics to safely deliver immunotherapies within tumors. These include nanobodies against two proven therapeutic targets—PD-L1 and CTLA-4. The drugs are continuously released by bacteria and continue to attack the tumor after just one dose, facilitating an immune response that ultimately results in tumor regression. The versatile probiotic platform can also be used to deliver multiple immunotherapies simultaneously, enabling the release of effective therapeutic combinations within the tumor for more difficult-to-treat cancers like colorectal cancer. The study is published today in Science Translational Medicine.
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CRISPR ‘minigene’ approach stops genetic liver disease in mice

A new CRISPR gene-editing technique prevented a genetic liver disease known to be driven by hundreds of different mutations and improved clinical symptoms in mice, Penn Medicine researchers reported in new proof-of-concept study published online in Science Advances. The findings suggest a promising CRISPR tool that could potentially treat patients with a rare metabolic urea-cycle disorder caused by a deficiency the enzyme, ornithine transcarbamylase (OTC), as well as other hereditary diseases triggered by different mutations on the same gene.
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Early treatment of schizophrenia may not slow disease progression

Stony Brook University-led study reveals that, despite the common view that early intervention in schizophrenia slows or stops mental decline, those who receive early intervention eventually experience the same declines as those whose treatment started later. The finding, published online in The American Journal of Psychiatry, suggests that studies of schizophrenia should take into account how long study participants have been symptomatic, otherwise treatments may appear more effective than they actually are.
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New drug leads could battle brain-eating amoebae

Brain-eating amoebae can cause particularly harmful forms of encephalitis, and more than 95% of people who develop these rare but devastating infections die. Despite the high mortality rate, there is currently no single effective drug available to fight these microbes. Now, however, researchers have designed some new compounds that show promise in the laboratory as treatments, according to a report in ACS Chemical Neuroscience.
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