CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers from the Perelman School of Medicine at the University of Pennsylvania ...
Nov 30, 2016
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Families of children with severe hemophilia A may want to take a fresh look at treatment options from human plasma. A study published in the New England Journal of Medicine on May 26 showed that participants who received ...
Jun 13, 2016
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Despite significant advances in hemophilia therapies and increased access to integrated treatment centers over the last half century, men with severe forms of this disease still experience physical limitations and disability, ...
Mar 16, 2016
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New research published online today in Blood, the Journal of the American Society of Hematology (ASH), identifies common genetic variants predominantly found in African Americans that double their risk for blood clots.
Feb 17, 2016
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Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and ...
Jan 20, 2016
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Biopharmaceuticals, or drugs that are based on whole proteins, are expensive to make and require refrigeration to store. Insulin, for example, is unaffordable and inaccessible to most of the global population.
Sep 30, 2015
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Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end. Their bodies lack the ability to make the clotting ...
Jul 24, 2015
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Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. ...
Mar 12, 2015
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The ability to pop a working copy of a faulty gene into a patient's genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine have devised a ...
Oct 29, 2014
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Two independent groups of researchers led by Sidney (Wally) Whiteheart, PhD, of the University of Kentucky, and Charles Lowenstein, MD, of the University of Rochester, have published important studies exploring the role that ...
Sep 18, 2014
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