When a couple loses a pregnancy, and especially when the same couple loses multiple pregnancies, doctors are often at a loss to explain why. For about 40 to 60 percent of couples with recurrent miscarriage (RM), the condition remains unexplained, even after costly testing. Chromosomal abnormalities — rearrangements of large chunks of DNA — in the genomes of one or…
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The award for scientific achievement was given to Dr. Rudolph Tanzi, the Joseph P. and Rose F. Kennedy Professor of Neurology at Harvard Medical School. Dr. Tanzi has spent his entire career focused on research related to identifying the genetic cause of Alzheimer’s disease. His doctoral thesis was on the discovery and isolation of the first Alzheimer’s gene – the amyloid precursor…
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Disc Medicine, headquartered in Cambridge, Massachusetts, announced the completion of a $50 million Series A financing. The round was led by Novo Holdings along with Access Biotechnology and founding investor Atlas Venture. The company also announced today that it had inked an exclusive deal with AbbVie for a series of hemojuvelin antagonist monoclonal antibodies. Disc Medicine is focused on hepcidin biology to restore red blood cell…
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It’s not every day that scientists get to publish a paper on Halloween about a skeleton, but that’s what the laboratories of Alan Brown in the Blavatnik Institute at Harvard Medical School and Rui Zhang at Washington University in St. Louis just did. As reported in the Oct. 31 print issue of Cell, the collaborating teams mapped out the atomic structures of significant components of…
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Promising Results Reported in Tay-Sachs Gene Therapy Trial

Researchers have obtained the first signs of clinical benefit in an early-stage gene therapy trial for Tay-Sachs disease, according to a presentation at the European Society of Gene & Cell Therapy (ESGCT) annual conference last week. Terence R. Flotte, MD, executive deputy chancellor, provost, and dean of the University of Massachusetts (UMass) School of Medicine, presented the results in Barcelona.…
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CAMBRIDGE, Mass. and DUBLIN, Oct. 30, 2019 /PRNewswire/ — Biogen Inc. (Nasdaq: BIIB) and Alkermes plc (Nasdaq: ALKS) today announced that the U.S. Food and Drug Administration (FDA) approved VUMERITY™ (diroximel fumarate), a novel oral fumarate with a distinct chemical structure, for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. Biogen holds the exclusive, worldwide license…
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Cancer evolution poses a central obstacle to cure, as resistant clones expand under therapeutic selection pressures. Genome sequencing of relapsed disease can nominate genomic alterations conferring resistance but sample collection lags behind, limiting therapeutic innovation. Genome-wide screens offer a complementary approach to chart the compendium of escape genotypes, anticipating clinical resistance. We report genome-wide open reading frame (ORF) resistance screens…
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Can Anna Feurer’s genes be used to help 10-year-old Avery Watts? Born with a genetic condition that results in life-threateningly high cholesterol, Watts travels twice a month to a Delaware hospital to have cholesterol filtered from her blood. Feurer, meanwhile, made headlines two years ago when scientists identified a mutation that protects her from heart disease by dramatically lowering levels…
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The investigational KRAS G12C inhibitor MRTX849 yielded clinical responses in patients with non-small cell lung cancer (NSCLC) and colorectal cancer harboring KRAS G12C mutations, according to data from a phase I clinical trial presented today at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics by Dana-Farber Cancer Institute researchers. Data demonstrating the efficacy of MRTX849 in preclinical studies and in two patients…
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CRISPR-Cas Genome Editing Applications for Disease Modeling and Cell Therapy. Click to download.><br />
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