Sarepta is best known for its RNA technology platform, which has led to two approved — though also controversial — drugs for Duchenne muscular dystrophy. Yet, Sarepta is also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing. Rare diseases have been an early target in…
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One in two men and one in three women will be diagnosed with cancer within their lifetimes. Fortunately, there has been significant progress in the treatment of a wide range of cancers, particularly when they are localized to a small area and can be removed with surgery or eliminated by treatments like chemotherapy. In some patients, though, the tumor cells…
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In many biomaterial systems, plastic deformation of extracellular matrix, degradation, or relaxation of stress are coupled and therefore their effects on cell phenotype are difficult to isolate. Using a polymer plasticizing architecture that specifically decouples irreversible creep from stress relaxation and modulus, network plasticity is shown to have a biphasic relationship with cell spreading.
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A hexanucleotide repeat expansion at C9ORF72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD). Initial studies of bacterial artificial chromosome (BAC) transgenic mice harboring this expansion described an absence of motor and survival phenotypes. However, a recent study by Liu and colleagues described transgenic mice harboring a large repeat expansion (C9-500) and reported decreased survival…
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UMass Medical School has received approval from the University of Massachusetts Board of Trustees for a project that will change the face of the Worcester campus and accelerate research into new therapeutics for some of the most challenging diseases that humans face. A new, nine-story biomedical research and education facility will be built on campus to support laboratory research growth and the…
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Early developmental specification can be modeled by differentiating embryonic stem cells (ESCs) to embryoid bodies (EBs), a heterogeneous mixture of three germ layers. Here, we combine single-cell transcriptomics and genetic recording to characterize EB differentiation. We map transcriptional states along a time course and model cell fate trajectories and branchpoints as cells progress to distinct germ layers. To validate this…
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An influential scientific journal’s list of top translational biotech researchers for 2019 includes Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases. Nature Biotechnology notes that its ranking of the top 20 biotech researchers is based on total patents…
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Researchers at Tufts University have found that those distinctly funky smells from cheese are one way that  fungi communicate with bacteria, and what they are saying has a lot to do with the delicious variety of flavors that cheese has to offer. The research team found that common bacteria essential to ripening cheese can sense and respond to compounds produced by…
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Á la CAR-T

The Chen Lab has identified a promising target for chimeric antigen receptor T (CAR-T) cell-based therapies for acute myeloid leukemia. CAR-T cell-based therapies have produced remarkable responses in patients with certain blood cancers, but translating that success to the treatment of other cancers has proven challenging. CAR-T cells are immune cells that have been engineered to home in on cancer…
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As cells grow, divide, and respond to their environment, their gene expression changes; one gene may be transcribed into more RNA at one time point and less at another time when it’s no longer needed. Now, researchers at MIT, Harvard, and the Broad Institute of MIT and Harvard have developed a way to determine when specific RNA molecules are produced…
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