Haiyan Zhou

Professor Haiyan Zhou serves as Professor of Genetic Medicine at University College London (UCL), affiliated with the Institute of Child Health and Department of Genetics and Genomic Medicine. She directs the UK Platform of Nucleic Acid Therapy (UPNAT Node) for rare diseases, co-leads the Therapeutic Innovation and Trials Community Domain in Genomics England, and acts as Deputy Theme Lead for Novel Therapies at the NIHR Great Ormond Street Hospital Biomedical Research Centre.

Prof. Zhou earned her MD-PhD in clinical medicine and skin pharmacology from Peking Union Medical College. Her research pioneers nucleic acid therapeutics (NAT) for rare genetic disorders, focusing on RNA-based approaches including antisense oligonucleotides, siRNA, and RNA editing for neuromuscular diseases. The Zhou Lab collaborates extensively across UCL with experts in muscular dystrophies, respiratory disorders, neurological conditions, and metabolic diseases to translate experimental therapies into clinical applications for pediatric patients.

Recent publications (2023-2025) reveal a dominant focus on optimizing RNA therapeutics for spinal muscular atrophy and collagen VI-related dystrophies, with key advances in allele-specific silencing, biomarker discovery, and preclinical-to-clinical translation. Her work consistently bridges molecular mechanisms with therapeutic development, emphasizing tissue-specific delivery and treatment response monitoring.

Scientific recognition includes:

• Harrington UK Rare Disease Scholar (2021)
• Lea Rose Spinal Muscular Atrophy Award (2017, 2015)
• Young scientist award from The SMA trust (2011)
• President Prize for the Young Myologist of the Year (World Muscle Society, 2006)

As principal investigator, Prof. Zhou secures funding from MRC, NIHR, and the Harrington Discovery Institute for RNA therapeutic development. She mentors early-career researchers through e-COST Action, Marie Skłodowska-Curie ITN, and UCL programs while supervising MSc/PhD students. Her leadership extends to directing UCL's MSc Personalized Medicine and Novel Therapies Programme and serving on multiple education committees.

The Zhou Lab operates as a multidisciplinary hub within UCL's Institute of Child Health, collaborating closely with the Dubowitz Neuromuscular Centre and international consortia like the Oligonucleotide Therapeutic Society. Current initiatives focus on individualized RNA therapy for pediatric rare diseases, with active pipelines for hereditary sensory neuropathy and COL6-related dystrophies.