Anew gene editing technique called prime editing, tested in human and mouse cells, rewrites DNA by only cutting a single strand to add, remove, or replace base pairs.
A combination of two experimental drugs appears to slow the decline of patients with amyotrophic lateral sclerosis, an illness often known as ALS or Lou Gehrig’s disease.
Within a mere eight years, CRISPR-Cas9 has become the go-to genome editor for both basic research and gene therapy. But CRISPR-Cas9 also has spawned other potentially powerful DNA manipulation tools that could help fix genetic mutations responsible for hereditary diseases.
Monica Coenraads had a terrible feeling something was wrong with her 14-month-old baby, Chelsea. She had not learned to walk. She had one word, duck, and then lost it.
Chelsea, it turned out, had a rare genetic disease, Rett Syndrome.
The University of Sheffield has been awarded the Queen’s Anniversary Prize for innovation in neuroscience
The prize is the highest national honour that recognises outstanding work by UK universities and colleges that demonstrate quality and innovation in their research
Sheffield has been recognised for improving patient outcomes for people living with neurodegenerative diseases such as Parkinson’s Disease and Motor Neurone Disease
A teenager is in a race against time to raise cash to help scientists find a cure for her rare disorder.
Maddi Thurgood, 15, from Ongar, is thought to be the only person in the UK and one of just 20 worldwide suffering from SPG15 (Spastic Paraplegia Gene 15).
The condition, a type of motor neuron disease, causes paralysis of the limbs, affects vision and hearing and can also cause juvenile Parkinson’s.
Maddi’s family have been contacted by the Gene Therapy Research team at the Sheffield Institute for Translational Neuroscience, one of the world leaders in this area of research.
Back in July, one of our users told us about Maddi Thurgood, a 16-year-old girl diagnosed with SPG15, and asked if we knew of any relevant data or contacts. SPG15 is a neurodegenerative disorder within the category of Hereditary Spastic Paraplegias.
A teenager is in a ‘cruel race against time’ to find a cure for her rare motor neuron disease before it’s too late.
Maddi Thurgood, 16, from Ongar, Essex, is believed to be the only person in Great Britain, and one of only 20 people in the world, to have Spastic Paraplegia Gene 15 (SPG15).