Public release date: 27-Feb-2012 [ | E-mail | Share ] Contact: Jennifer Santisi jsantisi@aaas.org 202-326-6213 American Association for the Advancement of Science A striking discovery in the study of human genetics was the focus of the most-visited press release posted on EurekAlert! in 2011. Researchers found that part of the non-African human X chromosome came from Neanderthals, confirming that they interbred with early human populations. Breakthroughs in mathematics, physics, chemistry, and medicine, along with discoveries in zoology, psychology, and astrobiology, were also featured in the 10 releases most often viewed by EurekAlert! readers in 2011. Website statistics collected over the course of the year identified the year's 10 most-visited news releases. The No. 1 most-visited press release garnered over 102,000 views. The (love) connection between early humans and Neanderthals described in 2011's most-visited news release Researchers from the University of Montreal and the CHU Sainte-Justine Research Center answered a nearly 10-year-old question concerning the origin of a part of the human X chromosome that shows characteristics different from the rest of the chromosome. Using the Neanderthal genome, which was sequenced in 2010, to compare with the human genome, researchers found a match. The mysterious piece of the human X chromosome's … Continue reading →

NOVATO, Calif., Feb. 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7.  MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS).  UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7.  MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.  su "Obtaining orphan drug designation for UX003 is a significant achievement for Ultragenyx that adds value to our development pipeline and attests to the importance of this product candidate in filling an unmet medical need," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We look forward to continuing to collaborate with Dr. Sly and colleagues at St. Louis University to advance this urgently needed therapy … Continue reading →

There is a tremendous opportunity in genetic medicine for innovation and for new players to make significant contributions, because it is still experimental, noted biologist and Nobel Laureate Dr David Baltimore said yesterday. “Today, it is mainly the province of biotechnology companies and universities, not big pharmaceutical companies,” he observed in a keynote presentation at the Qatar International Conference on Stem Cell Science and Policy 2012. There are new genetic tools available – though they are still experimental - to treat diseases which involve adding, subtracting or modifying genes in the cells of the body. “However, they are powerful tools and I am confident they will be an important part of the medicine of the future,” he said. Speaking on ‘The hematopoietic stem cell (HSC) as a target for therapy against cancer and Aids,’ Dr Baltimore explained that HSCs are one of the few cell types routinely used for bone marrow transplant. The HSCs are easily accessible, retroviruses can be used to carry genes into these stem cells, the genes are then expressed in all of cells that derive from the HSC and can correct inherited defects and bring genes that perform therapy under a programme called engineering immunity. “Though … Continue reading →

SUNRISE, Fla., Feb. 28, 2012 (GLOBE NEWSWIRE) -- Bioheart, Inc. (BHRT.OB) announced that the company will conduct the ANGEL trial using adipose (fat) derived stem cell technology or LipiCell(TM) at the University of Miami Miller School of Medicine. Bioheart recently applied to the FDA to begin trials using adipose derived stem cells in patients with chronic ischemic cardiomyopathy. "Dr. Joshua Hare and the University of Miami are world leaders in the field of stem cell research," said Mike Tomas, President and CEO of Bioheart. "We look forward to working with these acclaimed experts and bringing the LipiCell(TM) technology to patients in the U.S." The clinical protocol of the ANGEL trial is designed to assess the safety and cardiovascular effects of intramyocardial implantation of autologous adipose derived stem cells (LipiCell(TM)) in patients with chronic ischemic cardiomyopathy. Joshua Hare, MD, Director of the Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine is the principle investigator of the clinical program. The Interdisciplinary Stem Cell Institute was established to capitalize on pioneering work in the use of adult stem cells for the repair of malfunctioning human organs. The goal of the Institute is to find new treatments for heart … Continue reading →

NEW YORK & PETACH TIKVAH--(BUSINESS WIRE)-- BrainStorm Cell Therapeutics Inc. (OTCBB: BCLI.OB - News), a leading developer of adult stem cell technologies and therapeutics, announced today that the prestigious Nature Reviews Neurology, a Nature Publishing Group Journal, highlighted recently published preclinical research results indicating that stem cells, generated with Brainstorm’s NurOwn™ technology, provide hope for Huntington disease's patients. In the preclinical studies conducted by leading scientists including Professors Melamed and Offen of Tel Aviv University and originally reported in Experimental Neurology, patients' bone marrow derived mesenchymal stem cells secreting neurotrophic factors (MSC-NTF) that were transplanted into an animal model of Huntington disease showed therapeutic benefits. Addressing the role of these MSC-NTF cells in Huntington disease, Professor Daniel Offen explains, "the premise is that such cells can be transplanted safely into affected areas of the brain, and thereby serve as vehicles for delivering neurotrophic factors." Offen expressed his hope that this cell-based therapy may eventually progress to the clinic. BrainStorm is currently conducting a Phase I/II Human Clinical Trial for Amyotrophic Lateral Sclerosis (ALS) also known as Lou Gehrig’s disease at the Hadassah Medical center. Initial results have shown that Brainstorm’s NurOwn™ therapy is safe, does not show any significant treatment-related … Continue reading →

ScienceDaily (Feb. 27, 2012) — For the past decade, scientists have been pursuing cancer treatments based on RNA interference -- a phenomenon that offers a way to shut off malfunctioning genes with short snippets of RNA. However, one huge challenge remains: finding a way to efficiently deliver the RNA. Most of the time, short interfering RNA (siRNA) -- the type used for RNA interference -- is quickly broken down inside the body by enzymes that defend against infection by RNA viruses. "It's been a real struggle to try to design a delivery system that allows us to administer siRNA, especially if you want to target it to a specific part of the body," says Paula Hammond, the David H. Koch Professor in Engineering at MIT. Hammond and her colleagues have now come up with a novel delivery vehicle in which RNA is packed into microspheres so dense that they withstand degradation until they reach their destinations. The new system, described Feb. 26 in the journal Nature Materials, knocks down expression of specific genes as effectively as existing delivery methods, but with a much smaller dose of particles. Such particles could offer a new way to treat not only cancer, but … Continue reading →