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Alnylam files clinical trial application for alpha-1 liver disease, presents data at DDW

Alnylam Pharmaceuticals, Inc. has announced that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).

19 May 2015

Study finds similarity in clinical progression between human patients and Huntington's disease monkeys

Transgenic Huntington's disease monkeys show similarity to humans with Huntington's in their progressive neurodegeneration and decline of motor control, scientists from Yerkes National Primate Research Center, Emory University, report.

14 May 2015

Ground-breaking study characterizes early-stage Alzheimer's abnormalities in the intact brain

Alzheimer's disease (AD) is a neurodegenerative condition that strikes at the heart of what makes us human: the ability to think, to feel, to remember and to communicate with those around us. The tragedy is compounded by the fact that there is currently no cure, no treatment, and no diagnostic method capable of identifying Alzheimer's at its early stages.

6 May 2015

Scientists produce low-allergen, low anti-nutritional inhibitor soybean

In the United States, nearly 15 million people and 1 in 13 children suffer from food allergy. In Arizona alone, every classroom contains at least two children with a food allergy. Soybeans are one of the eight foods regulated by the Food Allergen Labeling and Consumer Protection Act, or FALPA. Soybean is a major ingredient in many infant formulas, processed foods and livestock feed used for agriculture. Soybeans contain several allergenic and anti-nutritional proteins that affect soybean use as food and animal feed.

5 May 2015

Rudolf Jaenisch honored with 2015 March of Dimes Prize in Developmental Biology

Rudolf Jaenisch, MD, who laid the groundwork for the development and use of induced pluripotent stem (iPS) cells - stem cells derived directly from adult tissue -- to potentially treat and cure a variety of human diseases, has received the 20th anniversary March of Dimes Prize in Developmental Biology.

30 Apr 2015

Ibrutinib (IMBRUVICA) may be effective for pancreatic ductal adenocarcinoma

Ibrutinib (IMBRUVICA) data presented yesterday by Pharmacyclics, Inc. at the American Association for Cancer Research Annual Meeting suggest that ibrutinib may be an effective therapeutic option for pancreatic ductal adenocarcinoma (PDAC), as shown in both a transgenic mouse model and an in-vivo model of patient-derived xenograft (PDX) mice (grafts of tissue taken from a pancreatic cancer patient and grafted into a mouse).

20 Apr 2015

CXCR3 molecule is key mediator of melanoma metastasis, shows research

In a unique partnership demonstrating excellence in "team science," Dartmouth investigators from Norris Cotton Cancer Center identified a role for the molecule CXCR3 (widely known to regulate the migration of immune cells) as a key mediator of melanoma metastasis.

7 Apr 2015

Pseudogenes may play role in cancer development, shows study

Pseudogenes, a sub-class of long non-coding RNA (lncRNA) that developed from the genome's 20,000 protein-coding genes but lost the ability to produce proteins, have long been considered nothing more than genomic "junk." Yet the retention of these 20,000 mysterious remnants during evolution has suggested that they may in fact possess biological functions and contribute to the development of disease.

3 Apr 2015

Iron oxide nanoparticles selectively target cell surface markers in tumor microenvironments

Nanoparticles hold great promise for cancer diagnostics and therapies, but only to the extent that they can be selectively guided to tumors and cancer cells.

30 Mar 2015

Broad Institute of MIT researcher honored for groundbreaking research using new gene editing tool

The newest gene editing tool - called CRISPR-Cas9 - is leading to new research possibilities in cell biology and neuroscience. This includes the ability to make transgenic animal models more quickly, which helps researchers better study diseases that affect thousands of people each year.

25 Mar 2015

Study reveals functional heterogeneity of CD4 T cells in immune systems

Utilizing a novel transgenic mouse model, Edward Usherwood, PhD of Dartmouth's Norris Cotton Cancer Center and collaborators found that CD4 T cells divide into two different populations that each has a different job. One type performs antiviral functions, and the other survives life in the host.

23 Mar 2015

Researchers identify promising new approach to treat pulmonary fibrosis

By uncovering the mechanism by which fibrous tissue cells in the lung multiply, researchers at The Saban Research Institute of Children's Hospital Los Angeles, along with colleagues in Mexico and Canada, have identified a promising new approach for the treatment of pulmonary fibrosis.

18 Mar 2015

Researchers reveal how malaria parasite deploys genetic trickery to escape immune system attack

Up to one million people -- mainly pregnant woman and young children -- are killed each year by the Plasmodium falciparum parasite, which causes the most devastating form of human malaria.

26 Feb 2015

Study identifies small loop in human prion protein that resists chronic wasting disease

Chronic wasting disease (CWD) — an infectious disease caused by prions — affects North American elk and deer, but has not been observed in humans. Using a mouse model that expresses an altered form of the normal human prion protein, researchers at University of California, San Diego School of Medicine have determined why the human proteins aren't corrupted when exposed to the elk prions.

24 Feb 2015

Study shows amyloid formation may link type 2 diabetes and Alzheimer disease

The pathological process amyloidosis, in which misfolded proteins (amyloids) form insoluble fibril deposits, occurs in many diseases, including Alzheimer disease (AD) and type 2 diabetes mellitus (T2D).

17 Feb 2015

Mice genetically deficient in serotonin are more vulnerable to social stressors

Mice genetically deficient in serotonin -- a crucial brain chemical implicated in clinical depression -- are more vulnerable than their normal littermates to social stressors, according to a Duke study appearing this week in the Proceedings of the National Academy of Sciences.

10 Feb 2015

Scientists potentially discover new therapeutic targets to halt progression of tumor cells

Chronic inflammation is directly associated with several types of cancer, yet the reasons as to why this happens at a cellular level remain unclear. Now, an international team of scientists led by researchers at The Wistar Institute has identified a multistep process showing not only how these cancers develop but also potentially discovering new therapeutic targets that could halt the formation and progression of tumor cells.

10 Feb 2015

Three Keck Medicine scientists of USC receive $4.3 million from CIRM for stem cell research

Three scientists from Keck Medicine of the University of Southern California have won grants exceeding $4.3 million from the California Institute for Regenerative Medicine for research that includes creating a temporary liver for transplant patients, finding novel ways to treat immune disorders and blood diseases and developing a new animal model for exploring diseases like heart failure, diabetes and neurodegenerative diseases.

5 Feb 2015

Scientists take a huge step forward in identifying root causes of psoriasis

Case Western Reserve scientists have taken a huge leap toward identifying root causes of psoriasis, an inflammatory skin condition affecting 125 million people around the world. Of the roughly 50,000 proteins in the human body, researchers have zeroed in on four that appear most likely to contribute this chronic disease.

22 Jan 2015

CUMC researcher develops new 3D microscope that can help view living things at very high speeds

Opening new doors for biomedical and neuroscience research, Elizabeth Hillman, associate professor of biomedical engineering at Columbia Engineering and of radiology at Columbia University Medical Center, has developed a new microscope that can image living things in 3D at very high speeds.

20 Jan 2015