ACADEMY PROGRAMME
Check the programme regularly for updates
Tuesday 26 August
14:00 - 15:00
Registration
15:00-15:15
Welcome
15:15-16:00
Hildegard Büning, Hannover Medical School
Adeno-Associated Virus (AAV) vectors as delivery tools in neuromuscular diseases
16:00-17:00
Introduction round and selection of the order of the student presentation + Coffee break
17:00-18:30 Student talks 1, 2 & 3
19:15
Dinner @ Le temps des cerises, 18, Rue de la Butte aux cailles
WARNING - They release the table at 19.30 so important to start on time
Wednesday 27 August
Group will arrive together by train from Gare de Lyon
10:00-11:00
Visit of Genethon
11:15-12:15
Visit of Yposkesi
12:30-13:30
Lunch at I-Stem building conference room
Mario Amendola, Genethon
Genome Editing and AAV Delivery for Utrophin-mediated Duchenne Muscular Dystrophy Therapy
13:30-14:30 Student talks 4 & 5
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14:30-15:00
Isabelle Richard, Genethon
AAV-mediated gene therapy in muscular dystrophies.
15:00-15:30
Guiseppe Ronzitti, Genethon
Opportunities and challenges in gene therapy for metabolic neuromuscular diseases
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15:30-17:00
Visit of I-STEM plus coffee break
17:00-18:00 Student talks 6 & 7​
18:00
Return to Paris
Free Evening in Paris
Thursday 28 August
09:00-09:30
Thierry VandenDriesche, VUBrussels
Development of CRISPR/Cas9-based gene editing for myotonic dystrophy type 1 (DM1): validation in patient-specific iPSC and DM1 mouse models
09:30-10:30 Student talks 8 & 9
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10:30-11:00
Coffee Break
11:00-11:30
Thierry VandenDriesche, VUBrussels
Turbocharging gene therapy for genetic muscle diseases: boosting tissue-targeted transcription in skeletal muscle, heart and diaphragm
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11:30-12:30 Student talks 10 & 11​
12:30-14:00
Lunch
14:00-14:30
Ana Buj Bello, Genethon
Gene therapy of myotubular myopathy: from POC studies to a clinical trial and more
14:30-15:00
Federic Relaix,
Rat Duchenne muscular dystrophy models for preclinical studies and deciphering tissue repair mechanisms
15:00-16:00 Student talks 12 & 13​
16:00-16:30
Coffee Break
16:30-17:30
Serge Braun, AFM Telethon
One can change the world with pancakes and balloons
18:30-
Surprise social event and dinner
Rue Saint Louis en l'île
Friday 29 August
09:00-09:45
Gloria González-Aseguinolaza, CIMA, University of Navarra, Pamplona
Between academia and industry: juggling roles to deliver solutions to patients
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09:45-10:15
Capucine Trollet, Institut de Myologie
Cell and Gene Therapy Approaches for Oculopharyngeal Muscular Dystrophy
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10:15-10:45
Arnaud Klein, Institut de Myologie
Tackling the RNA toxicity: gene therapy approaches for Myotonic Dystrophy Type 1​
10:45-12:30
Visit i-Motion
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11:45- 12:15
Alexandre Mejat