Scientists comment on top line results from a phase I/II trial of a gene therapy to slow the progression of … read more

A study published in The New England Journal of Medicine looks at a new CRISPR gene therapy for children with a rare … read more

Scientists react to news of MHRA approving gene therapy to cure sickle-cell disease.   Dr Rashid Kazmi, Honorary Senior Lecturer … read more

Researchers use gene therapy – via a lentiviral vector – for patients with beta-thalassaemia, in a new study, published in the NEJM. read more

Scientists have published a new study in Nature which reports the successful use of the CRISPR-Cas9 genome editing technique as a tool to correct hearing loss, in a mouse model of deafness.

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A new study, published in PNAS, reports the use of gene therapy to improve visual function in a mouse model of retinal degeneration. read more

In a new study published in Molecular Therapy researchers – using gene immunotherapy – were able to demonstrate the prevention and reversing of the symptoms of multiple sclerosis (MS) in mice. read more

In a new study published in Nature Communications scientists report gene therapy improving dystrophic symptoms, for over two years, in a canine model of Duchenne muscular dystrophy. read more

New research published in Nature Communications looks at ways of using the genome editing tool CRISPR to restore full-length dystrophin in mouse models of Duchenne muscular dystrophy. read more

Two papers publishing in Nature Biotechnology have reported – in a mouse model of Usher syndrome – the efficient delivery of genes to the inner ear, which led to an unprecedented correction of hearing loss and balance symptoms. read more

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

In work to be presented at the American Society of Haematology annual meeting in December scientists have described their work using gene editing techniques in attempt to treat a patient with relapsed acute lymphoblastic leukaemia. It is thought to be the first time this technique has been attempted in a person with leukaemia. read more

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

Publishing in the Journal of Endocrinology, scientists have used a form of gene therapy with the aim of reducing weight in rats. They report that rats which were treated with therapy which delivered the gene for the protein leptin lost weight, while rats which had a control treatment gained weight. read more

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 (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

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

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

It has been reported that a research group in China has used the CRISPR genome editing technique to modify human embryos with a specific genetic default. Reports suggest that the scientists used non-viable fertilised embryos with only limited success in achieving the attempted modification. read more

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