by University College London Credit: CC0 Public Domain Gene therapy has partly restored the function of the retina’s cone receptors in two children who were born completely color blind, reports a new study led by University College London (UCL) researchers. The findings, published in Brain, provide hope that the treatment is effectively activating previously dormant communication...
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord responsible for voluntary movements and muscle control. In a new study, published in the journal Theranostics, researchers at University of California San Diego School of Medicine report that a gene therapy approach, developed at UC...
by Medizinische Hochschule Hannover Graphical abstract. Credit: Molecular Therapy (2022). DOI: 10.1016/j.ymthe.2022.07.003 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, for spinal muscular atrophy (SMA), a congenital neuromuscular disease with severe muscle...
by Daegan Miller, University of Massachusetts Amherst Lila Gierasch (center), professor of biochemistry and molecular biology at UMass Amherst. Credit: UMass Amherst In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently...
by University of California, Los Angeles Credit: CC0 Public Domain A new study in mice finds that a gene therapy developed by a UCLA researcher appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and recurrent seizures. The treatment potentially could represent an improvement upon...
by A’ndrea Elyse Messer, Pennsylvania State University Bioprinting during surgery of bone constructs used as a controlled gene co-delivery platform for the repair of skull defects. Credit: Dong Heo, Kyung Hee University; Ozbolat Lab, Penn State / Penn State Given enough time and energy, the body will heal, but when doctors or engineers intervene, the...
by Dennis Thompson An experimental cream-based gene therapy may soon become the first U.S. government-approved means for treating a rare and devastating skin disease that produces “butterfly children.” Patients with recessive dystrophic epidermolysis bullosa (EB) are called butterfly children “because their skin is as fragile as the wings of a butterfly,” explained lead researcher Dr....
by Bob Yirka, Medical Xpress Graphical abstract. Credit: New England Journal of Medicine (2022). DOI: 10.1056/NEJMoa2113708 An international team of researchers has found more evidence that a new gene therapy may help some people with hemophilia A. In their paper published in the New England Journal of Medicine, the group describes the characteristics of the clinical trial and...
by Mike Silver, Tufts University Illustration of a lipid nanoparticle surrounded by plasma proteins. Credit: Min Qiu Tufts researchers are building a reputation for precision targeting in drug delivery. Their tools: tiny lipid-based nanoparticles (LNPs) fine-tuned to latch on to specific tissues, organs, even cell types within the body. Their latest creation: LNPs that carry...
NORWEGIAN UNIVERSITY OF SCIENCE AND TECHNOLOGY “Many millions of elderly people worldwide suffer from sarcopenia, a disease that is characterized by muscle wasting. A large proportion become so frail that they can no longer exercise,” says Jose Bianco Moreira, a researcher at NTNU, the Norwegian University of Science and Technology. “Gene therapy is the most...
