Gene therapy

The history of the study of the gene

The gene: an intimate history by Siddhartha Mukherjee…

Developing gene therapy to treat cystic fibrosis: challenges and successes

More than 25 years after the gene for cystic fibrosis was discovered, there are signs that correcting this genetic deficit could soon be possible.…

‘One-size-fits-all’ approach will not work for drug evaluation and funding

Evaluation and funding of the most promising and innovative medicines will require a separate approach to other drugs in the future, according to the chair of the government-commissioned accelerated access review.…

Advanced therapy medicinal products present challenges for pharmacists

Few pharmacists have experience handling advanced therapy medicinal products (ATMPs) — medicines based on genes, cells or tissue engineering — but the field is likely to grow and presents special challenges for pharmacists.…

Gene therapy for ‘bubble boy’ syndrome on track for EU approval

Children with an extremely rare immune deficiency disease are set to have access to gene therapy, following a recommendation for approval by the European Medicines Agency.…

Researchers unravel function of HIV defence protein

Researchers investigate why TRIM5α protein lost ability to degrade HIV in rhesus monkey study.…

Gene editing used to treat muscular dystrophy in mice

CRISPR/Cas9 system injected into muscle or bloodstream restored function of dystrophin gene, marking an important step in gene therapy research.…

Genetically engineering humans: a step too far?

Among scientists and health professionals there are differing opinions on how far we should take genetic engineering. Robert Sparrow and Glenn Cohen discuss the merits and dangers of using this technology beyond disease treatment and prevention.…