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expert reaction to mouse study of gene therapy for Alzheimer’s disease

A group of scientists have described their use of a specific gene therapy to treat Alzheimer’s disease in a mouse model. Publishing in the journal Proceedings of the National Academy of Sciences the scientists report that the upregulation of the PGC-1α gene brought about reduced biological markers of the disease and improved function in the mice. read more

gene-edited immune cells used in human patient with leukaemia – a case study

A team at Great Ormond Street Hospital (GOSH) and the UCL Institute of Child Health has used the TALENs gene editing technique to modify immune T-cells, in attempt to treat a patient with relapsed acute lymphoblastic leukaemia. TALENs (Transcription Activator-Like Effector Nucleases) are molecular tools that act like scissors, cutting specific gene sequences. In this case, scientists used TALENs to engineer immune T-cells to target leukaemia cells. This is the first time this technique has been attempted in a person with leukaemia. read more

expert reaction to results from gene therapy trial for cystic fibrosis

Genetic disorders underlie many diseases, and gene therapies have the potential to alleviate the severity of some conditions or even cure them. Researchers publishing in the The Lancet Respiratory Medicine journal have detailed their use of a gene therapy to treat cystic fibrosis, reporting a modest and variable effect compared to placebo. read more

cystic fibrosis gene therapy trial results

Cystic fibrosis (CF) is the most common lethal inherited disease in the UK, affecting around 10,000 people nationally and over 90,000 worldwide. Patients’ lungs become filled with thick sticky mucus and they are vulnerable to recurrent chest infections, which eventually destroy the lungs. The cause of CF, mutations in a gene located on chromosome 7, was identified in 1989, opening the door to introducing a normal copy of this gene using gene therapy. The UK Cystic Fibrosis Gene Therapy Consortium, a group of scientists and clinical teams from Imperial College London, the Universities of Oxford and Edinburgh, Royal Brompton & Harefield NHS Foundation Trust and NHS Lothian, have been working together to develop a gene therapy since 2001. read more

gene editing of human embryos

As some in science circles have been predicting, gene-editing techniques which are already widely used in plants and animals have now been applied to human embryos. In what is thought to be a world first, Chinese scientists used CRISPR/Cas9 to modify the DNA of human embryos, thereby attracting global headlines about science fiction becoming science fact, as well as warnings about slippery slopes and designer babies. read more

expert reaction to paper on genome editing of mitochondria in mice

Researchers have attempted to fix errors in mitochondrial DNA, which lead to a range of disorders, by using genome editing techniques. Publishing in the journal Cell, they report the use of the techniques in mice and suggest that they might in future be used as a therapeutic alternative to mitochondrial donation. read more

new gene therapy for heart failure

Heart failure is a debilitating condition that affects more than 750,000 people in the UK, but there is currently no treatment capable of improving heart function once the disease takes hold. Experts from the British Heart Foundation came to the SMC to talk about the beginning of clinical trials for a new gene therapy approach to treatment for advanced heart failure. read more

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