Plenary program

Plenary program

 Tuesday 3 September 2019  
de Doelen
Welcome Lunch
Grote Zaal
Chair: A. van der Ploeg (Rotterdam, The Netherlands)

Grote Zaal
Plenary Session 1: Building Bridges

Chairs: J. Smeitink (Nijmegen, The Netherlands), H. Michelakakis (Athens, Greece), G. Salomons (Amsterdam, The Netherlands)
14.30-15.00Organoids as models of human disease
Hans Clevers (Utrecht, The Netherlands)
15.00-15.30The role of DNA damage repair and transcription stress in aging and the impact of nutrition
Jan Hoeijmakers (Rotterdam, The Netherlands)
15.30-16.00The lysosome as a control center of cell metabolism
Andrea Ballabio (Naples, Italy)

Grote Zaal
Plenary Session 2: RNA based therapy and Gene therapy
Chairs: P. Pijnappel (Rotterdam, The Netherlands), F. Mingozzi (Philadelphia, USA)
16.30-17.00AAV gene therapy for inherited diseases
Alberto Auricchio (Naples, Italy)
17.00-17.30HSC gene therapy for neurometabolic and neurodegenerative disorders
Alessandra Biffi (Cambridge, USA)
17.30-18.00Opportunities and challenges for RNA therapy development
Annemieke Aartsma Rus (Leiden, The Netherlands)

Grote Zaal
Chair: E. Morava (Rochester, USA)
Urea cycle disorders: of ammonia, dialysis and future directions of management
Johannes Häberle (Zurich, Switzerland)
 Wednesday 4 September 2019  

Grote Zaal
Plenary Session 3: Regenerative medicine
Chairs: G. Schaaf (Rotterdam, The Netherlands), J. Häberle (Zurich, Switzerland)
11.00-11.30Hurdles in successful examples of stem cell-based regenerative medicine
Graziella Pellegrini (Modena, Italy)
11.30-12.00Ex vivo gene editing studies with human hepatocytes from patients with urea cycle defects
Stephen Strom (Stockholm, Sweden)
12.00-12.30Human induced pluripotent stem cells come of age in modeling cardiovascular disease
Christine Mummery (Leiden, The Netherlands)

Grote Zaal
Plenary Session 4: Mitochondrial disorders
Chairs: J. Smeitink (Nijmegen, The Netherlands), C. Dionisi-Vici (Rome, Italy)
16.30-17.00Moving towards clinical trials for mitochondrial disease
Shamima Rahman (London, United Kingdom)
17.00-17.30The Pursuit of Precision Mitochondrial Medicine: Harnessing pre-clinical and animal models of mitochondrial disease
Marni J. Falk (Philadelphia, USA)
17.30-18.00Mitochondria in Alzheimer disease: It’s not what you think
Eric A Schon (New York, USA)
 Thursday 5 September 2019  

Grote Zaal
Plenary session 5: Metabolomics / System biology
Chairs: G. Ruijter (Rotterdam, The Netherlands), V. Kozich (Prague, Czech Republic)
09.00-09.30Modeling human metabolism in health and disease
Ines Thiele (Galway, Ireland)
09.30-10.00Innovative analytical tools for metabolomics-driven systems biology to investigate monogenetic and complex disorders
Thomas Hankemeier (Leiden, The Netherlands)
10.00-10.30Integrative network biology approaches to understand rare inborn errors of metabolism
Carmen Argmann (New York, USA)
 Friday 6 September 2019  

Grote Zaal
Plenary Session 6: Lysosomes-Innovative insights
Chairs: A. van der Ploeg (Rotterdam, The Netherlands), G. Parenti (Naples, Italy)
09.00-09.30Disorders of lysosome-related organelle biogenesis
Marjan Huizing (Bethesda, USA)
9:30-10:00Challenges and concepts to combat diseases causes by deficiencies of lysosomal hydrolases and membrane proteins
Paul Saftig (Kiel, Germany)
10:00-10:30The future of therapies for Lysosomal disorders, lessons from the past
Simon Jones (Manchester, United Kingdom)

Grote Zaal

Chair: Gajja Salomons (Rotterdam, The Netherlands)
From fatty acid oxidation to riboflavin - Make metabolites great again!
Christine Vianey-Saban (Lyon, France)