Friday 11 September
08:30-09:00
Registro
09:00-11:00
AAV viruses as delivery vehicles for gene editing tools
Modera:
09:00 Nerea Zabaleta, Cima, Pamplona
INV01 AAV viruses and generation of viral vectors for gene editing (single/dual AAV approach vs LNP)
09:20 César Trigueros, Viralgen, Donosti
INV02 AAV manufacturing
09:40 Lluis Montoliu, CNB
INV03 Gene editing approaches and novel nucleases
10:00 Paula Rio, Ciemat, Madrid
INV04 DNA repair machinery, CRISPR off-targets and safety
10:20 Carmen Unzu, Cima, Pamplona
INV05 Immune responses to AAV vectors and gene editing elements
10:40 Gloria Gonzalez-Aseguinolaza, Cima, Pamplona
INV06 Preclinical development and regulatory considerations
11:00-11:30
Coffee Break
11:30-13:30
Using AAV vectors for different gene editing applications
Modera:
Preclinical research of four different AAV-based gene editing strategies under development in Spain
11:30 Gloria Gonzalez-Aseguinolaza, Cima, Pamplona
INV07 Gene editing for primary hyperoxaluria
12:00 Carmen Unzu, Cima, Pamplona
INV08 Gene editing for hereditary hearing loss
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12:30 Nerea Zabaleta, Cima, Pamplona
INV09 Gene editing for polycystic kidney disease
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13:00 Francisco Martin, Genyo, Granada
INV10 Gene editing for epigenetic modulation of hematopoietic stem cell differentiation
13:30-14:30
Lunch
14:30-16:00
Gene editing design workshop
Students are divided in small working groups to design a therapy for a proposed indication based
on information that is provided at the beginning of the activity. They should design not only the strategy
but also the preclinical development towards a clinical trial.