As we age, our bodies tend to develop diseases like heart failure, kidney failure, diabetes, and obesity, and the presence of any one disease increases the risk of developing others. Traditional drug development targets only ...
Nov 4, 2019
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182
Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which once would require a bone marrow ...
Oct 18, 2019
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154
Researchers at UC Santa Cruz have demonstrated a new, rapid method to obtain donor stem cells for bone marrow transplants using a combination of Viagra and a second drug called Plerixafor.
Oct 10, 2019
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133
A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large ...
Oct 7, 2019
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In experiments with rats, pigs and monkeys, Johns Hopkins Medicine researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and effective in humans, the technique could provide a ...
Sep 30, 2019
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284
A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers.
Sep 19, 2019
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41
A group of scientists and clinicians from the UK, the Netherlands, Italy and the U.S. have worked together and made an important discovery that explains an often fatal organ defect that affects unborn babies and children.
Sep 16, 2019
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26
Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic ...
Aug 21, 2019
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334
Building on a track record of developing adeno-associated viral (AAV) vectors as a groundbreaking clinical tool for gene therapy and gene editing, Children's Hospital of Philadelphia (CHOP) researchers report a more sensitive ...
Jul 30, 2019
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82
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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