Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.
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A team led by scientists at The Scripps Research Institute (TSRI) has found how to boost or inhibit a gene-silencing mechanism that normally serves as a major controller of cells' activities. The discovery could lead to a powerful new class of drugs against viral infections, cancers and other diseases.
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RNA, once considered a bit player in the grand scheme by which genes encode protein, is increasingly seen to have a major role in human genetics.
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RNA molecules, made from DNA, are best known for their role in protein production. MicroRNAs (miRNAs), however, are short (~22) nucleotide RNA sequences found in plants and animals that do not encode proteins but act in gene regulation and, in the process, impact almost all biological processes - from development to physiology to stress response.
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RNAi-based therapeutics company Benitec Biopharma Limited today announced the selection of the University of California, San Diego, Health Sciences as the second site for its upcoming phase I/II first-in-man trial for TT-034 in Hepatitis C infections.
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New classes of drugs that can silence specific genes, such as small interfering RNAs (siRNAs), offer great therapeutic potential. But the specific delivery of siRNAs to target cells to exert their effects remains a significant challenge. A novel nanoparticle-based approach that enables more efficient delivery of siRNA drugs is presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.
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Autophagy, the process by which cells that are starved for food resort to chewing up their own damaged proteins and membranes and recycling them into fuel, has emerged as a key pathway that cancer cells use to survive in the face of assault by chemotherapy and radiation.
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Deborah E. Wiley, Chair of The Wiley Foundation, John Wiley & Sons, Inc. (NYSE: JWa & JWb) today awarded the 2013 Wiley Prize in Biomedical Sciences to Dr. Michael Young, Rockefeller University, Dr. Jeffrey Hall, Brandeis University (Emeritus), and Dr. Michael Rosbash, Brandeis University.
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Most cancer treatments are blunt. In an attempt to eradicate tumors, oncologists often turn to radiation or chemotherapy, which can damage healthy tissue along with the cancerous growths.
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Researchers at the University of North Carolina at Chapel Hill have discovered that a protein found in the cells surrounding pancreatic cancers play a role in the spread of the disease to other parts of the body.
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Scientists are trying to engineer apples so that the most widely consumed fruit in Europe no longer triggers allergic reactions.
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Alnylam Pharmaceuticals a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).
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An antidepressant drug used since the 1960s may also hold promise for treating sickle cell disease, according to a surprising new finding made in mice and human red blood cells by a team from the University of Michigan Medical School.
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Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced today that that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells for bone marrow protection in the treatment of glioblastoma multiforme.
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The new study published in Cancer Discovery, the flagship journal of the American Association of Cancer Research (AACR), involving three Spanish and six American research centres, presents significant results in treating cancer patients with nanoparticles containing ribonucleic acid interference (RNAi) molecules.
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Deborah E. Wiley, Chair of The Wiley Foundation, John Wiley & Sons, Inc., announced today that the twelfth annual Wiley Prize in Biomedical Sciences will be awarded to Dr. Michael Young, Rockefeller University, Dr. Jeffrey Hall, Brandeis University (Emeritus), and Dr. Michael Rosbash, Brandeis University.
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The Damon Runyon Cancer Research Foundation, a non-profit organization focused on supporting innovative early career researchers, named 15 new Damon Runyon Fellows at its fall Fellowship Award Committee review.
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Scientists have wrestled to understand why Huntington's disease, which is caused by a single gene mutation, can produce such variable symptoms. An authoritative review by a group of leading experts summarizes the progress relating cell loss in the striatum and cerebral cortex to symptom profile in Huntington's disease, suggesting a possible direction for developing targeted therapies.
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Scientists from the Florida campus of The Scripps Research Institute (TSRI) have uncovered a group of what have been considered relatively minor regulators in the body that band together to suppress the spread of cancer from its primary site.
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Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office has issued Notices of Allowance for three patent applications from the company's exclusively held Tuschl II patent estate.
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