UK Platform of Nucleic Acid Therapy for rare disease treatment (UPNAT) Node

The UPNAT node, part of Rare Disease Research UK (RDR UK), is building a roadmap for rapid bench-to-bedside translation of Nucleic Acid Therapies for patients with Rare Disease in the UK, by creating a national network including scientists, clinicians, geneticists, patient advocates and charities, industrial partners, international non-profit organizations and regulatory bodies.