T-cell therapy fights viral infections following stem cell transplants
January 13, 2023 -- 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
Optimizing AAV vectors for gene therapy delivery
January 10, 2023 -- Adeno-associated viruses (AAV) can be engineered to deliver DNA to target cells. It is used extensively for the development of viral vector-based gene therapies in the pipeline. However, researchers encounter challenges with producing sufficient quantities of AAVs for gene therapy products, and they struggle to get delivery vehicles and their genetic payloads into the brain across the blood-brain barrier. Read More
Discovery of destructive CRISPR enzyme opens diagnostic, therapeutic applications for molecular scissors
January 4, 2023 -- 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
January 4, 2023 -- 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
December 16, 2022 -- 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
December 14, 2022 -- 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
Machine learning used to encode commands for immune cells
December 9, 2022 -- Researchers at University of California San Francisco (UCSF), in collaboration with a team at IBM Research, have developed a virtual molecular library of thousands of “command sentences” for cells, based on combinations of “words” that guide engineered immune cells to seek out and kill cancer cells without pausing. Read More
Shenandoah expands cytokine, growth factors portfolio for cell and gene therapies
December 7, 2022 -- Shenandoah Biotechnology, a Fujifilm Irvine Scientific company, has announced the expansion of its CTGrade portfolio of cytokine and growth factors for cell and gene therapies that are manufactured following current good manufacturing practices. Read More
CIRM awards $8M for gene therapy research on rare, incurable diseases
December 2, 2022 -- The California Institute for Regenerative Medicine (CIRM) has awarded $8 million in grants for two research teams investigating gene therapy for rare and incurable diseases. Read More
Scientists uncover viral vectors that cross the blood-brain barrier
November 23, 2022 -- 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
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