by Scott Lafee, University of California – San Diego PET scan of a human brain with Alzheimer’s disease. Credit: public domain Researchers at University of California San Diego School of Medicine, with colleagues elsewhere, have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer’s disease (AD), a key step toward eventually...
by Gail McCormick, Pennsylvania State University Credit: CC0 Public Domain A new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other rare diseases. The method, developed and tested in...
IOS PRESS Amsterdam, April 6, 2021 – The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, in a broader spectrum of patients in order to obtain expanded data on its side effects...
MASSACHUSETTS GENERAL HOSPITAL BOSTON – Researchers have used a genetic engineering strategy to dramatically reduce levels of tau–a key protein that accumulates and becomes tangled in the brain during the development of Alzheimer’s disease–in an animal model of the condition. The results, which come from investigators at Massachusetts General Hospital (MGH) and Sangamo Therapeutics Inc., could lead...
UNIVERSITY COLLEGE LONDON An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells has been cleared by the body. The research team monitored five patients who were successfully cured...
by Bob Yirka , Medical Xpress Credit: CC0 Public Domain A team of researchers affiliated with multiple institutions in Germany has developed a type of gene therapy that could be used to help people with cystic fibrosis. In their paper published in the journal Nature Nanotechnology, the group describes how they developed a new class of non-viral gene...
Amber Tong Senior Editor Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45. We just have to wait until 2024 to find...
Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer’s disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia. The protein,...
Danielle Ternes/BioPharma Dive AUTHOR Ned Pagliarulo@NedPagliarulo PUBLISHED Feb. 12, 2021 The toxic globules of sugar and fat slowly pile up in the body’s cells, accumulating in blood vessels and in major organs like the kidney and heart. Typically, special-purpose proteins within the cell would tear apart and break down the toxins. But for people with...
by Children’s Hospital of Philadelphia Credit: CC0 Public Domain Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently used in gene therapy trials for those conditions. The vector, an engineered vehicle for delivering functional...
