PTAC/CFTR

S16--The Promise of Nucleic Acid-Based Therapies in CF: Progress & Prospects

Chair(s)

• 

  Deepika Polineni

  Washington University in St. Louis
• 

  Patrick Harrison

  Division of Pulmonary Medicine, Cincinnati Children’s Hospital, Cincinnati, Ohio, USA

This session will explore innovative strategies to enhance gene therapy for cystic fibrosis (CF). The focus will be on developing optimal gene constructs by utilizing shortened and optimized sequences, advanced codon usage, and other strategies to improve delivery, function, and expression. Additionally, the session will cover cutting-edge techniques for enhancing delivery systems to ensure therapeutic genes effectively reach target tissues. Novel nucleic acid-based therapies (NABT) offer the best hope for curative therapy for all pwCF and encompass a large number of strategies to restore functional CFTR, including gene therapy with a variety of viral vectors, splice-switching oligonucleotides (SSOs), and mRNA-based approaches. Progress and barriers in development of NABT will be discussed, including the biology of quiescent cells and immune response to NABT.

Presentations:

• 2:30 PM - 4:30 PM PDT

  S16.1- Progress on antisense oligonucleotide therapies in cystic fibrosis
  

  Speaker: Batsheva Kerem, PhD – Hebrew University
• 2:30 PM - 4:30 PM PDT

  S16.2- Lentiviral gene therapy – the journey into the clinic
  

  Speaker: Eric Alton, MA, MD, FRCP, FHEA, FERS, FMedSci – National Heart and Lung Institute, Imperial College London
• 2:30 PM - 4:30 PM PDT

  S16.3- Editing the CFTR gene
  

  Speaker: Patrick T. Harrison – Division of Pulmonary Medicine, Cincinnati Children’s Hospital, Cincinnati, Ohio, USA
• 2:30 PM - 4:30 PM PDT

  S16.4- Real-world challenges & perspectives
  

  Speaker: Deepika Polineni – Washington University in St. Louis