Nancy A. Melville November 06, 2023 PHOENIX — Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research...
Tag: <span>Gene Therapy</span>
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Researchers pursue three gene therapies for rare inherited disease
by Catherine Caruso, Harvard Medical School Credit: Pixabay/CC0 Public DomainWhen neurobiologist David Corey showed up at a rare disease conference in 2017, he had no idea that he would enter a race against time to develop a treatment for it. The conference was for Usher syndrome type 1F. Patients with this condition have a gene mutation...
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Researchers design gene therapy that can effectively target glioblastoma
by Brigham and Women’s Hospital Glioblastoma (histology slide). Credit: Wikipedia/CC BY-SA 3.0Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months. Immunotherapies, which mobilize the body’s immune defenses against a cancer, have not been effective for GBM, in part because the tumor’s surrounding environment...
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Gene therapy study identifies potential new treatment for liver cancer
by UC Davis Graphical Abstract. Credit: Molecular Therapy (2023). DOI: 10.1016/j.ymthe.2023.04.019 Gene therapy that induces the body to create microRNA-22 (miR-22), a naturally occurring molecule, successfully treated mice with hepatocellular carcinoma, the most common form of liver cancer. The miR-22 treatment also reduced liver inflammation and produced better survival outcomes with no observable toxicity compared...
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Study shows promise of gene therapy for alcohol use disorder
OREGON HEALTH & SCIENCE UNIVERSITY A form of gene therapy currently used to treat Parkinson’s disease may dramatically reduce alcohol use among chronic heavy drinkers, researchers at Oregon Health & Science University and institutions across the country have found. The study in nonhuman primates showed that implanting a specific type of molecule that induces cell growth effectively...
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Research sheds new light on gene therapy for blood disorders
by University of Michigan Credit: National Institutes of Health Research from experts at Michigan Medicine, the Children’s Hospital of Philadelphia and Penn Medicine is breaking ground on new ways of treating blood disorders, such as sickle cell anemia, through gene therapy. The study is published in the journal Science. To cure blood disorders, patients must undergo high dose chemotherapy...
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Gene therapy hope for children with kidney disease
by University of Bristol Image [left to right]: Professor Moin Saleem, Dr Wen Ding [Academic Clinical Lecturer in Paediatric Renal Medicine] and Professor Gavin Welch [Professor of Renal Cell Biology] from the University of Bristol’s Medical School Credit: Kidney Research UK Researchers at the University of Bristol have made a remarkable step forward in finding a potential cure for...
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Gene therapy treats chronic pain by dialing down sodium
NEW YORK UNIVERSITY IMAGE: AN ARTISTIC REPRESENTATION OF THE INTERACTION BETWEEN THE NAV1.7 SODIUM ION CHANNEL AND COLLAPSIN RESPONSE MEDIATOR PROTEIN 2 (CRMP2). THE RESEARCHERS IDENTIFIED A UNIQUE REGULATORY SEQUENCE IN NAV1.7 THAT IS REQUIRED FOR NAV1.7 FUNCTION. THEY FOUND THAT THIS PEPTIDE DISRUPTED THE INTERACTION WITH CRMP2 AND REDUCED EXCITABILITY IN SENSORY NEURONS. THE...
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Gene therapy eyedrops restored a boy’s sight. Similar treatments could help millions
by LAURA UNGAR and FREIDA FRISARO Dr. Alfonso Sabater, checks Antonio Vento Carvajal’s eye under a blue light after applying a stain to check to see if more ulcers had developed, before a gene therapy treatment, Thursday, July 6, 2023, at University of Miami Health System’s Bascom Palmer Eye Institute in Miami. Antonio was born...
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Gene Therapy Promising for Reversal of Hereditary Vision Loss
Liam Davenport July 13, 2023 An unapproved gene therapy for Leber hereditary optic neuropathy (LHON) led to a marked improvement in the eyesight of patients with a severe, progressive form of the disease who received the therapy as part of an early access program. Results of a study of more than 60 patients who received lenadogene nolparvovec...