Session Details

• Jaap Jan Zwaginga, MD, PhD, Leiden University, Netherlands

• Marina Cavazzana, MD, PhD, Département de Biothérapie- Hospital Necker-Enfants Malades, Assistance Publique–Hôpitaux de Paris, Centre d’Investigation Clinique en Biothérapie, INSERM VMR1163, Imagine Institute, Université Paris Cité, France - Gene Therapy for SCD: Achievements and issues to solve
• Johan Flygare, MD, PhD, Lund University, Sweden - Single-cell-multiomics demonstrates molecular efficacy of a clinical lentiviral vector for gene therapy of RPS19-deficient DBA
• Frank J.T. Staal, PhD, Leiden University Medical Center, Netherlands - Curative stem cell based gene therapy for RAG-deficient SCID

Session Objectives

1. To understand the principles (and limits) of stem cell based gene therapy and for what inherited diseases it might provide a life long cure.
2. To learn the current clinical translation of this type of therapy by 3 challenging examples: for Hemoglobinopathies, for Blackfan Diamond and for Severe Immune-deficiencies
3. To oversee– if proven to be successful – the hurdles for further implementation of these therapies and compare specifics for large patient groups like Hemoglobinopathies vs what is additional problematic for rare patient groups.
4. To discuss collaborative solutions, requirements and resolve because indeed morbidity and mortality of severe inherited disorders could be changed into a life long cure.