Haiyan Zhou
University College London
Research areas: Biochemistry, Genetics and Molecular Biology · Molecular Biology · Muscle Physiology and Disorders · Neurogenetic and Muscular Disorders Research · Amyotrophic Lateral Sclerosis Research
0000-0001-8440-3074
About
Dr Haiyan Zhou is a Professor of Genetic Medicine at University College London (UCL) and a UK Harrington Rare Disease Scholar. Her research specializes in the preclinical development and clinical translation of nucleic acid therapies (NAT) for rare genetic disorders. Prof. Zhou is the Director of the UK Platform for Nucleic Acid Therapy (UPNAT), a specialized initiative for rare diseases funded by the UK Medical Research Council (MRC) and the NIHR. She co-leads the Genomics England Research Network Community on Therapeutic Innovations and Trials. As the principal investigator on a range of research projects, she is at the forefront of developing RNA-based therapeutics for a wide spectrum of genetic disorders. She serves as the Deputy Theme Lead for the Novel Therapy Theme at the NIHR Biomedical Research Centre at UCL Great Ormond Street Hospital (GOSH BRC), where she leads the Individualized RNA Therapy Program for paediatric rare diseases. In addition to her research, Prof. Zhou directs the MSc in Cell, Gene and Novel Therapies at UCL, where she plays a key role in training the next generation of scientists in cutting-edge therapeutic development.
Employment
-
University College London Professor of Genetic Medicine2024 - Present
-
University College London Associate Professor2021 - 2024
-
University College London Lecturer (Assistant Professor)2018 - 2021
Education
Education history is unavailable.
Projects & Funding
-
Proof-of-concept RNA-editing studies in muscular dystrophies Great Ormond Street Hospital Charity2026 - 2027
-
Developing RNA editing as a therapeutic strategy for nonsense mutation-associated Muscular Dystrophy Muscular Dystrophy UK2026 - 2030
-
Preclinical Study of a Novel RNA-targeted Therapy for SPTLC1-Related Hereditary Sensory Neuropathy Harrington Discovery Institute, University Hospitals2025 - 2027
-
Preclinical studies of antisense oligonucleotides therapy in a humanized mouse model of SPTLC1-related hereditary sensory neuropathy Rosetrees Trust2025 - 2027
-
Preclinical development of peptide- ASO therapy for COL6-related muscular dystrophy LifeArc2025 - 2028
-
A European Training Program to Foster the Full Therapeutic Potential of Antisense Technology across Tissues UKRI2024 - 2028
-
Establishing a UK platform of nucleic acid therapy for rare disease UKRI2023 - 2028
Publications (24)
- Nucleic Acid Therapeutics in Rare Disease: From Precision Medicine to Programmable Platforms Save
- From N-of-1 therapy to health-system implementation Save
- Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies Save
- RNA‐Based Therapies for Inherited Metabolic Disorders Save
- Genetic therapies for neurological diseases Save
- Prenatal Therapy Rescues Neurodevelopmental Defects Associated with Spinal Muscular Atrophy Save
- Antisense oligonucleotides reverse SPTLC1-related hereditary sensory neuropathy in a mouse model Save
- Characterization of severe COL6-related dystrophy due to the recurrent variant COL6A1 c.930+189C>T Save
- Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseases Save
- Morpholino-Mediated Exon Inclusion for Spinal Muscular Atrophy (SMA) Save
- Recombinant SMN protein synergizes with spinal muscular atrophy therapy to counteract pathological motor neuron phenotypes Save
- Profiling neuroinflammatory markers and response to nusinersen in paediatric spinal muscular atrophy Save
- Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing Save
- Consistent Delivery of Adeno-Associated Virus via Lateral Tail-Vein Injection in Adult Mice Save
- A pH-responsive cetuximab-conjugated DMAKO-20 nano-delivery system for overcoming K-ras mutations and drug resistance in colorectal carcinoma Save
- Report of the European Medicines Agency Conference on RNA-Based Medicines Save
- Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics Save
- Microvasculopathy in spinal muscular atrophy is driven by a reversible autonomous endothelial cell defect Save
- Response of plasma microRNAs to nusinersen treatment in patients with SMA Save
- Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion Save