Huntington's disease (HD) is a rare disease characterized by abnormal chorea movement and caused by the Huntingtin (Htt) gene mutation and neurodegeneration in a brain area called the striatum. A research group led by Dr. ...
Feb 28, 2020
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Researchers at Indiana University School of Medicine are looking for ways to heal wounds by using a healing protein that is active in fetuses, but largely inactive in adults and absent in diabetic adults.
Nov 2, 2022
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A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD).
Sep 25, 2019
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Researchers led by a team at Massachusetts Eye and Ear have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types—the most commonly used viral vectors for in vivo gene therapy. AAVs are ...
Jan 23, 2020
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It was when Xinglong Wang, PhD, received a call from a desperate father of a middle-aged son with amyotrophic lateral sclerosis (ALS) that he realized the extraordinary importance of his work. "Can you help save my son's ...
Jan 23, 2017
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Ana Buj Bello's team, a researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert's myotonic dystrophy, the most common neuromuscular ...
Jun 25, 2019
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An experimental mRNA vaccine provides protection in preclinical animal models against infection from Borrelia burgdorferi, the bacteria that causes Lyme disease, according to new research from the Perelman School of Medicine ...
Sep 19, 2023
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In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of ...
Mar 23, 2017
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Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...
Mar 30, 2018
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Gene therapies aim to cure severe, barely treatable monogentic diseases caused by a defect in a single gene. Medical hopes are correspondingly high. Some gene therapies have already been approved in Europe—for example, ...
Aug 2, 2022
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