BOSTON – For diagnosing nonalcoholic steatohepatitis (NASH), liver biopsy is "the reference standard," Dean Hum, president of Genfit Corp., told BioWorld. "I'm not going to call it the gold standard." Some of the reasons for denying biopsies a gold sticker are obvious. Biopsies are invasive, which makes them risky, expensive and loathed by patients and doctors alike. "In the real world, doctors don't always do biopsies – many say they very rarely do biopsies unless other data points in several directions and they need that for clarity," Gail Cawkwell told BioWorld.
Researchers at Harvard Medical School and The Banner Alzheimer's Institute have identified a potential protective variant that appears to have delayed the onset of clinical symptoms by several decades in one individual with a presenilin mutation. Presenilin mutations cause an autosomal dominant, early onset form of AD that usually leads to clinically noticeable cognitive impairments in one's 40s.
A subtype of schizophrenia is related to abnormally high brain levels of hydrogen sulfide (H2S), which has important implications for the development of new treatments, according to a study by researchers at the RIKEN Center for Brain Science (CBS) in Japan.
A Chinese study has identified small-molecule compounds that can selectively reduce levels of the mutant Huntingtin (HTT) protein involved in the pathogenesis of Huntington's disease (HD), and possibly other neurodegenerative diseases involving mutant protein accumulation.
BOSTON, Mass. – KRAS used to be the poster child for undruggability. No more. Amgen Inc. and Mirati Therapeutics Inc. are in the clinic with KRAS inhibitors. Boehringer Ingelheim GmbH is planning to join them by the end of the year. And multiple other companies are moving programs forward.
Chinese researchers have developed the first green tea-triggered genetic control system for future gene- and cell-based precision medicine applications, and then used it to treat diabetes in mice and monkeys, they reported in the Oct. 23, 2019, issue of Science Translational Medicine.
Two very different roles were reported for the protein REST last week. In adults, REST activation appeared to extend lifespan by reducing overall brain activity. Principal investigator Bruce Yankner, professor of genetics at Harvard Medical School and co-director of the Paul F. Glenn Center for the Biology of Aging, told BioWorld that in postmortem brain samples of individuals who had had no cognitive impairments at the time of their death, his team found "a correlation between down-regulation of excitation and extended longevity."
Australian scientists have discovered promising new candidate analgesic molecules derived from a Penicillium fungus, which represents a promising resource for the development of safer new analgesics, they reported in the Oct. 14, 2019, edition of Proceedings of the National Academy of Sciences (PNAS).
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