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New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK

New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK

Reading Time: 2 minutes This article was written by Dr Keith Mayl and Dr Ahmad Al Khleifat of King’s College London. Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for patients affected by a specific genetic form of ALS, the most common type of MND. ALS is a…

Tofersen: antisense oligonucleotide drug shows promising results in Phase 1/2 trial

Tofersen: antisense oligonucleotide drug shows promising results in Phase 1/2 trial

Reading Time: 3 minutes A recent press release by the pharmaceutical company Biogen reported preliminary results from an ongoing clinical trial investigating a form of precision therapy in people with SOD1-related MND. This drug, known as tofersen, is now in the final stages of Phase 1/2 testing in centres across the world, including Sheffield in the UK. Tofersen is…

Making antisense of RNA editing

Reading Time: 3 minutes Association-funded PhD student, Helena Chaytow (Royal Holloway, University of London), is using DNA to develop a targeted treatment for MND. Now entering her final year, we report on what she’s achieved so far and her future plans. The background Helena’s research is looking at the chemical messenger ‘glutamate’. Glutamate is released by motor neurones in…

SOD1 Stuff

Reading Time: 4 minutes With all the talk of new gene discoveries in recent years, the Sunday morning scientific session returned to the original discovery in 1993 that mutations in the SOD1 gene were responsible for around a fifth of familial (inherited) MND cases and 2-3% of all cases of the disease. Although much of our understanding of MND…

Antisense seems to make sense

Reading Time: 3 minutes Results from a phase I clinical trial of a drug known as ISIS 333611 have been published open-access online in the scientific journal Lancet Neurology on 29 March 2013. This is the first time researchers have tested the effects of delivering an antisense oligonucleotide directly into the human cerebral spinal fluid (the fluid between the spinal…