Genomics
Newborn screening for neuromuscular disorder leads to improved outcomes in Australian study
Newborn screening for spinal muscular atrophy (SMA) improves outcomes by enabling timely access to disease-modifying therapy, a prospective study published in the Lancet Child & Adolescent Health found. Read More
T-cell therapy fights viral infections following stem cell transplants
Baylor College of Medicine and Washington University School of Medicine researchers studied posoleucel, an investigational off-the-shelf T-cell therapy that simultaneously targets six different viruses. The results, published January 11 in the journal Clinical Cancer Research, showed promising antiviral efficacy and safety in a phase II clinical trial of patients who had undergone stem cell transplantation to treat blood diseases including cancer. Read More
Discovery of destructive CRISPR enzyme opens diagnostic, therapeutic applications for molecular scissors
The discovery of a CRISPR enzyme that destroys DNA and RNA in target cells has unlocked opportunities to use molecular scissors in diagnostics and to selectively destroy diseased cells. Read More
Cancer-killing vaccine may also prevent brain cancer
Harvard Stem Cell Institute scientists are harnessing a new method of turning cancer cells into potent, anti-cancer agents. Their new cell therapy approach to eliminating established tumors also trains the immune system to prevent cancer from recurring, providing long-term immunity. The NIH-funded study, published January 4 in Science Translational Medicine, showed promising results when the approach was tested on mice with glioblastoma, a deadly brain cancer. Read More
Common genetic cause of late-onset ataxia revealed
A Quebec-led international collaboration has discovered a previously unknown common genetic cause of late-onset cerebellar ataxia. The study, published December 14 in the New England Journal of Medicine, may potentially improve diagnosis and open new treatment avenues for thousands of people with this debilitating neurodegenerative condition worldwide. Read More
CRISPR system targets toxic RNA to improve Huntington's in mice
Genome-editing CRISPR technology has reduced the toxic RNA that drives Huntington's disease in mice, providing preclinical proof of principle for a new way to treat the fatal neurodegenerative disorder, according to a study published on December 12 in Nature Neuroscience. Read More
Scientists uncover viral vectors that cross the blood-brain barrier
Using a "directed-evolution" strategy in mice and macaques, scientists at the Broad Institute of Massachusetts Institute of Technology and Harvard have identified adeno-associated viruses that cross the blood-brain barrier, advancing efforts to develop neurological disease gene therapies. Read More
Multiplexed 'CAR Pooling' accelerates evaluation of anticancer cell therapy receptors
Researchers have developed a new method for comparing massive numbers of CAR T cells, each with slightly different molecular features, to determine which is most effective and long-lasting against cancer. Read More
Cell & Gene Meeting on the Mesa 2022 Recap
The Science and Medicine Group team was onsite at this year's Cell & Gene Meeting on the Mesa getting the who, what, and when on the product announcements, science sessions, and deals taking place this year. Find out what we learned in our recap. Read More
Gene therapy targets overactive brain cells in mice, potential neurological treatment
University College London researchers have developed a gene therapy for neurological and psychiatric diseases that reduces the excitability of overactive brain cells. Tested in mice, the scientists contend the treatment could potentially be used in human brain diseases caused by excessive activity of a small number of brain cells. Read More
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