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Monday, 06.04.2020

09:00-12:00  

Workshops 
Public engagement: Paula Rio
Grant proposals and funding: Anne Galy
Presentation skills: Renee Watson

12:00-13:00 Lunch for those attending the spring school
13:00-14:30 Hildegard Büning (ESGCT) & George Vassilopoulos (HSGTRM)
Welcome address
Keynote:
INV01 Nathalie Cartier
Gene Therapy for neurodegenerative diseases from genetic to complex diseases : delivery and targets
 
14:30-15:50 Session 1: Spotlight on delivery tools
INV02 Axel Schambach, Hannover Medical School
What you should know about retro and lentiviral vectors
INV03 Hildegard Büning, Hannover Medical School
AAV - a little virus rocks gene therapy
15:50-16:20 Coffee Break
16:20-17:40 Session 2: Spotlight on delivery tools II
INV04 Aglaia Athanassiadou
Non-viral gene transfer
INV05 Anne Galy
Innate and adaptive immune responses
17:40-18:40 Keynote:
INV06 Toni Cathomen
Risk assessment of gene editing associated off-target effects
19:30-22:00 Meet the expert dinner at the hotel

Tuesday, 07.04.2020

09:00-10:20
 
Session 3: The payload
INV07 Zoltan Ivics, Paul Elrich Institute, Langen
Wide awake and ready to move: non-viral genome engineering with Sleeping Beauty transposon vectors
INV08 Alberto Auricchio Tigem, Naples
How to transfer large genes with a small vector?
10:20-10:50 Coffee break
10:50-12:10 Session 4: Once you have produced...
INV09 Eduard Ayuso,
Université de Nantes
Nucleic acids contaminants in AAV stocks: methods for quantification and strategies to reduce their presence
INV10 Rebecca Matsas
Modelling brain disease: advancing from animal models to human iPSCs-based organoids.
12:10-13:10 Keynote:
INV11 Juan Bueren, CIEMAT/Ciberer, Madrid
Moving from basic stem cell research to clinical gene therapy?
13:10-14:30 Lunch
14:30-15:30  Keynote: 
INV12 Fatima Bosch 
Gene therapy approaches for Mucopolysaccharidosis.
 
15:30-16:50  Session 5: Gene therapy for the liver 
INV13 Federico Mingozzi, Genethon, Paris
Liver gene transfer for the systemic delivery of therapeutic proteins
INV14 Gloria Gonzalez Aseguinolaza, FIMA, Pamplona
Targeting the second largest organ in the human body to fix inherited diseases
16:50-17:20 Coffee break
17:20-18:40   Session 6: Immunotherapy and cancer
INV15 Vincenzo Cerullo, University of Helsinki
Dressing viruses in tumor’s clothing: welcome to the cloning-free oncolytic vaccine era
INV16 Eugenio Montini, San Raphaele, Milan
Cancer immunotherapy. How to boosts our natural defenses to fight cancer
19:00-22:30 Dinner

Wednesday, 08.04.2020

09:00-10:20   Session 7: Gene and cell therapy for the CNS
INV17 Françoise Piguet, ICM Paris
Gene therapy strategy for CNS
INV18 Terry Flotte,  University of Massachusstts, Amherst
Clinical Translation of AAV gene therapy for CNS: Tay-Sachs and ALS
10:20- 11:10 Coffee Break
11:10-12:30   INV19 Alessandro Aiuti
TBC
INV20 Stylianos Michalakis
Retinal gene therapy
12:30-13:30 Lunch
13:30 - 14:50 Session 8: Gene therapy for primary immuno-deficiencies
INV21 Claire Booth, UCLondon
Evolution of gene therapy for PID and wheredo we go next?
INV22 Giuliana Ferrari, SR Tiget
The engineering of hematopoietic stem cells to treat genetic blood disorders
14:50-16:00 Closing: Hildegard Büning and George Vassilopoulos

Extraordinary General Assembly of the ESGCT