- Gene Therapy
- Cell Therapy
- Genome Editing
- +۳ مورد دیگر
Roles and Affiliations: Rafael J. Yáñez-Muñoz is a Professor of Advanced Therapy and Director of the Centre of Gene and Cell Therapy at the Department of Biological Sciences, Royal Holloway University of London. He holds expertise in gene and cell therapy, particularly for neurodegenerative and inherited diseases such as ataxia telangiectasia and spinal muscular atrophy. Education: BSc and PhD in Biochemistry and Molecular Biology from the Autonomous University of Madrid. Previously held Lecturer positions at King’s College London and University College London. Research Interests: Focuses on developing safer gene therapy methods using episomal vectors and genome editing (e.g., CRISPR-Cas). Key areas include non-integrating lentiviral vectors, viral vector modification, and treatments for rare diseases. His lab (AGCT) explores therapies for spinal muscular atrophy, spinal injury, Parkinson’s disease, and primary immunodeficiencies. Publications and Impact: Over 85 publications, including seminal work on non-integrating lentiviral vectors and gene editing. Notable contributions to Gene Therapy as Editor-in-Chief and leadership roles in the British Society for Gene and Cell Therapy. Grants and Collaborations: Principal Investigator on projects funded by the SMA Trust, BBSRC, MRC, and others. Collaborates with institutions like Genethon (France) and Harvard University. Key initiatives include the UK SMA Research Consortium and CHASE-IT for spinal injury therapy. Awards and Roles: Trustee and Chair of Genetic Alliance UK, President of the British Society for Gene and Cell Therapy (2021–2025). Recognized for advocacy in rare disease awareness and policy, including contributions to the UK National Strategy for Rare Diseases. Advisees and Teams: Supervised numerous PhD students and postdocs, including Sahar Akbari Vala, Melika Fard, and Ellie Chilcott. Active in mentoring and lab management, with a focus on training the next generation of gene therapy researchers. Labs and Outreach: Leads the Advanced Gene and Cell Therapy (AGCT) lab, hosting annual Rare Disease Day events to raise awareness. Engages in public education through lectures, media interviews, and initiatives like the SMA Trust fundraising cycle events.
