REVOLUTIONIZING THERAPIES FOR CHRONIC DISEASES

Concurrent Speaker(s)

• 

  Claire Booth, MBBS MSc PhD FRCPCH (she/her/hers)

  Infection, Immunity and Inflammation Department
  UCL Great Ormond Street Institute of Child Health, United Kingdom

Presenting Author(s)

• 

  Alice Del Vecchio

  Molecular Medicine
  Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Italy
• 

  Rosario Isasi, JD MPH (she/her/hers)

  Human Genetics
  University of Miami, United States
• 

  Mo Li, PhD

  Bioscience
  King Abdullah University of Science and Technology (KAUST), Saudi Arabia
• 

  Sarah Saietz, MSc, BSc (she/her/hers)

  Department of Clinical Science, Intervention and Technology (CLINTEC)
  Karolinska Institutet, Sweden

This session delves into groundbreaking advancements in gene therapies, focusing on the hematopoietic stem cell (HSC) system. Topics include gene addition and editing techniques, including CRISPR/Cas9 and prime editing, and their applications in treating genetic diseases affecting the blood, central nervous system (CNS), skeletal systems, and more. This session will also cover emerging in vivo gene therapy strategies targeting HSCs and immune cells, as well as the regulatory, accessibility, and economic challenges associated with these advanced therapies.

Presentations:

• “THE REGULATORY SYSTEM IS CAPABLE OF HANDLING MOST OF THE SCARY SCENARIOS THAT PEOPLE THROW UP”: KEY INFORMANT PERSPECTIVES ON PRENATAL GENE EDITING
  

  Presenting Author: Rosario Isasi, JD MPH (she/her/hers) – University of Miami
• DEVELOPMENT AND PRECLINICAL VALIDATION OF CELLTHRPE1: A STEM CELL-DERIVED RETINAL THERAPY FOR DRY AGE-RELATED MACULAR DEGENERATION
  

  Presenting Author: Sarah Kieler Saietz, MSc, BSc (she/her/hers) – Karolinska Institutet
• CRISPR-CAS9-INDUCED DOUBLE-STRAND BREAKS DISRUPT MAINTENANCE OF EPIGENETIC INFORMATION
  

  Presenting Author: Mo Li, PhD – King Abdullah University of Science and Technology (KAUST)
• ENGINEERED HUMAN TUBULES REVEAL PATHOPHYSIOLOGICAL MECHANISMS OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
  

  Presenting Author: Alice Del Vecchio – Istituto di Ricerche Farmacologiche Mario Negri IRCCS
• THE PRESENT AND FUTURE OF GENE THERAPIES FOR INBORN ERRORS OF IMMUNITY
  

  Concurrent Speaker: Claire Booth, MBBS MSc PhD FRCPCH (she/her/hers) – UCL Great Ormond Street Institute of Child Health