BioNews
Vector
A delivery system used in the laboratory to carry foreign DNA into a cell. Modified viruses are commonly used as vectors in gene therapy.
Spotting the zebras among the horses: Understanding metabolic disorders
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...
New approach to delivering gene therapy for cystic fibrosis
A lipid nanoparticle-based gene therapy for cystic fibrosis restores biological function in a preclinical model...
First patient treated with gene therapy to improve heart bypasses
A heart attack patient has become the first person to be treated in a clinical trial of an experimental gene therapy, which aims to strengthen blood vessels after coronary bypass surgery...
Pioneering gene therapy for Hunter syndrome shows clinical success
A UK toddler with a rare genetic condition was the first person to receive a new gene therapy that appears to halt disease progression...
First evidence that gene therapy can slow Huntington's disease
A microRNA treatment is the first therapy to show evidence of slowing Huntington's disease progression....
DNA-coated nanostructures enhance CRISPR delivery and precision
A new nanostructure has been developed that could enable CRISPR genome-editing components to enter human cells more efficiently and safely...
Genome editing research targets late-onset Tay Sachs
Base editing has been used to correct the mutation causing late-onset Tay-Sachs disease in mice and human cells...
Rare immune disease treated successfully with personalised gene therapy
A world-first gene therapy has successfully been used to restore the immune system of a teenager with a rare genetic condition...
