A lone genetic mutation can cause a life-changing disorder with effects on multiple body systems. Lysosomal storage diseases, for example, of which there are dozens, arise due to single mutations that affect production of ...
Jul 29, 2020
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In an unprecedented effort, hundreds of thousands of researchers and clinicians worldwide are locked in a race against time to develop cures, vaccines, and better diagnostic tests for COVID-19, the illness caused by the virus ...
Apr 24, 2020
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Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that delivers therapeutic genes—for use in gene therapy for sickle cell disease. In advanced lab tests ...
Oct 2, 2019
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Ethiopia's second largest city, Dire Dawa, is dealing with a chikungunya fever outbreak. Around 20,000 cases of the mosquito-borne viral infection have been reported since the outbreak started in March 2019. This is the second ...
Sep 16, 2019
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It is a remarkable proof for the concept of IOB: working closely hand in hand, our molecular and clinical researchers have developed a library of 230 adeno associated viral vectors (AAVs), each with a different synthetic ...
Jul 19, 2019
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Some people live much longer than average, partly thanks to their DNA. A study, published in the European Heart Journal, shows that it could be possible to replicate this genetic gift even for those lacking it. The way is ...
Jul 10, 2019
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Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment ...
Feb 1, 2019
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UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX. The gene therapy, which was tested ...
Jan 11, 2019
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Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function, found researchers from the Perelman School of Medicine at the University of Pennsylvania ...
Oct 18, 2018
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A new gene therapy for treating an inherited form of Amyotrophic Lateral Sclerosis has been given an Orphan Drug designation by the European Medicines Agency. The therapy is already in preclinical development at EPFL.
May 23, 2018
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