The UK Rare Disease Research Platform has been established with a £14 million investment by the Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR), with the aim of accelerating rare disease research to improve diagnosis and treatment. Comprised of  11 specialist research nodes the Platform brings together expertise across the UK. 
The Nucleic Acid Therapies node - UPNAT is lead by TransNAT investigator Dr Haiyan Zhou, and 
includes Prof Francesco Muntoni and Prof Nick Lench, also TransNAT partners. 
UK Platform of Nucleic Acid Therapy for rare disease treatment - UPNAT - Rare Disease Research UK (rd-research.org.uk)

https://rd-research.org.uk/

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