Gene therapy and genetic pharmacology prospects and challenges for rare diseases
Contents
Moderator
Jingying Wu
Mentor for PhD Students, Physician
Departments of Neurology and Genetics & Rare Diseases, The Sixth People’s Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China
Departments of Neurology and Genetics & Rare Diseases, The Sixth People’s Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China
Dr. Wu is a clinician-scientist focused on clinical and translational research in neurogenetics and neuroimmunology. She has published 10 papers as first author or co-first author in high-impact journals, including Science, Movement Disorders, and Translational Neurodegeneration, and holds a national patent. As first author in Science, she reported a breakthrough demonstrating that replacement of pathogenic microglia can effectively halt disease progression in adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in both animal models and patients.
Dr. Wu has been recognized with several honors, including the Outstanding Postdoctoral Fellow Award from the National Neuroscience Young Scientists Forum and selection for the Youth Talent Promotion Program of the Chinese Society of Neurology.
Dr. Wu has been recognized with several honors, including the Outstanding Postdoctoral Fellow Award from the National Neuroscience Young Scientists Forum and selection for the Youth Talent Promotion Program of the Chinese Society of Neurology.
Speaker
Daniel Scherman
Editor-in-Chief, Rare Disease and Orphan Drug Journal
General Director, Foundation for Rare Diseases (FFRD – Fondation Maladies Rare)
Scientific Secretary, International Rare Disease Research Consortium (IRDiRC)
Head, Medicine and Life Sciences Division, European Academy of Science
Corresponding Member, French Académie nationale de Pharmacie
General Director, Foundation for Rare Diseases (FFRD – Fondation Maladies Rare)
Scientific Secretary, International Rare Disease Research Consortium (IRDiRC)
Head, Medicine and Life Sciences Division, European Academy of Science
Corresponding Member, French Académie nationale de Pharmacie
Prof. Scherman has long been committed to bridging basic research and clinical translation. His work spans molecular biology, genetics, nanomaterials, and biomedical engineering, with particular emphasis on gene editing, RNA interference, nanomedicine delivery, and cancer diagnostics and therapy. As of 2025, he has authored over 400 publications indexed in Scopus, with an H-index of 80 and more than 28,000 citations. His contributions have been recognized with numerous prestigious awards, including the Emilia Valori Prize from the French Academy of Sciences, the Neurobiology Prize from the French National Academy of Medicine, the Silver Medal of the French National Centre for Scientific Research, and the Ordre des Palmes Académiques.
