A delivery system used in the laboratory to carry foreign DNA into a cell. Modified viruses are commonly used as vectors in gene therapy.
A six-year-old girl has died following an experimental genome editing treatment, prompting an investigation into the clinical trial by the university involved...
An experimental epigenome-editing therapy has shown a favourable safety profile and increased lean muscle mass in patients with a rare muscular dystrophy in an early-stage trial...
A new experimental gene therapy has shown early success in treating a rare genetic epilepsy syndrome...
Adeno-associated virus gene therapy vector DNA has been found integrated within tumour tissue in a patient with Hurler syndrome who had been treated years earlier...
A long-term clinical trial of a gene therapy for a rare genetic form of deafness has demonstrated sustained hearing restoration, while a separate gene therapy for the same condition has received approval from the Food and Drug Administration...
Metabolic disorders can seem forbiddingly complex, but a simplified approach to explaining their biological basis can lead to improved understanding, earlier diagnosis and better management, argues Dr Srividya Sreekantam...
A lipid nanoparticle-based gene therapy for cystic fibrosis restores biological function in a preclinical model...
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