PTAC/CFTR
Patrick Harrison, PhD
University College Cork
Anna Cereseto
University of Trento
This symposium shows how recent developments in gene editing and gene delivery are helping the progression towards genomic correction of CF lung disease. All four talks will combine some level of overlap between editing and delivery. The main focus of two of the talks is describe the use of a number of gene editing strategies to permanently correct different CF-causing mutations, their effect on restoring CFTR function, and options to further improve them. The other two talks focus more on delivery options and reaching different target cells in the lung. The approaches described in this session show strong basic sciences underpins the therapeutic pipeline for everyone.
Speaker: Anna Cereseto – University of Trento
Speaker: Matthew Porteus, MD, PhD – Stanford University
Speaker: Marianne Carlon, PhD (she/her/hers) – KU Leuven
Speaker: Stephany Tzeng, PhD – Johns Hopkins University