Genetics
First gene therapy for Tay-Sachs disease successfully given to two children
Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development.
Feb 15, 2022
0
2
Page 30 - News tagged with gene therapy
Diseases, Conditions, Syndromes
Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French ...
Feb 10, 2022
0
3
Neuroscience
Experimental gene therapy targets Duchenne muscular dystrophy
Children in Rochester were recently among the first in the nation to receive an experimental treatment for Duchenne muscular dystrophy (DMD). The study is part of an accelerating trend of clinical trials involving gene therapies ...
Feb 10, 2022
0
20
Neuroscience
Star-like brain cells display unique activity patterns
The way people experience the world occurs due to complex and intricate interactions between neurons in the brain. Now, a study, published 9th February 2022 in Science Advances, suggests that astrocytes—star-shaped, non-neuronal ...
Feb 9, 2022
0
143
Oncology & Cancer
Doctors: Long-lasting cell treatment cured leukemia patients
In 2010, doctors treated Doug Olson's leukemia with an experimental gene therapy that transformed some of his blood cells into cancer killers. More than a decade later, there's no sign of cancer in his body.
Feb 6, 2022
0
2
Genetics
Tweaked genes borrowed from bacteria excite heart cells in live mice
Biomedical engineers at Duke University have demonstrated a gene therapy that helps heart muscle cells electrically activate in live mice. The first demonstration of its kind, the approach features engineered bacterial genes ...
Feb 4, 2022
0
72
Genetics
Gene therapy promotes transfusion independence for severe beta-thalassemia
A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in the New England ...
Feb 4, 2022
0
24
Medications
Dexamethasone enhances transgene expression
Dexamethasone, a glucocorticoid with anti-inflammatory and immunosuppressive effects, can transiently increase the expression of therapeutic genes delivered using adeno-associated virus (AAV) vectors. A new study, which showed ...
Feb 1, 2022
0
0
Genetics
Gene therapy for the treatment of Huntington's disease
Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington's disease after a single administration. An adeno-associated virus (AAV) gene ...
Jan 25, 2022
0
166
Medical research
Calcium: Important not just for your bones but also for your heart
Researchers from Osaka University found a previously unknown gene mutation that can cause an incurable heart condition called dilated cardiomyopathy. This gene, BAC5, is important for the movement of calcium ions in the heart ...
Jan 19, 2022
0
73
