A change in the sequence of DNA. Mutations occurring in genes may prevent them from working properly, which might result in disease (a genetic condition). Mutations occur naturally at a low rate, but can also be induced by radiation and some chemicals.
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 new CRISPR-based approach has been designed to selectively eliminate human cells with disease-causing genes in vitro...
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...
A child born with a congenital retinal disorder has had her sight restoredby an NHS-funded gene therapy...
Breakthrough Prizes in Life Sciences have been awarded to two teams of scientists for their work on gene therapies for inherited blood and blindness conditions...
A routine genetic test used by the NHS to guide prescription of a common chemotherapeutic drug now includes a genetic variant more prevalent in people of African descent...
A newly described neurodevelopmental disorder has been linked to changes in a small noncoding gene...
New regulations in sperm donation are being implemented by Belgium's Federal Agency for Medicines and Health Products, following revelations regarding the use of sperm carrying a cancer-causing mutation, and widespread breaches of donor limits...
Combining genome sequencing and cellular reprogramming has allowed researchers to dissect the genetic causes of a recently discovered rapid-ageing disorder...
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