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  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01--Nucleotide-Based Therapeutics: Delivery & Gene Editing
    Location: Theater B (Exhibit & Poster Hall)
    Chair: Patrick Harrison – University College Cork
    Chair: Shingo Suzuki, PhD (he/him/his) – University of Alabama at Birmingham
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.1- Modulating nucleolin trafficking can significantly enhance DNA nanoparticle-mediated gene expression in the lung
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Matthew Siefert, M.S. – Cincinnati Children's Hospital Medical Center
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.2- Formulating viral vectors with hypertonic saline enhances airway gene transfer
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Ashley Cooney, PhD – University of Iowa
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.3- Intratracheal administration of AAV2.5T-SP183-fCFTRΔR in combination with doxorubicin is able to correct the mucociliary clearance defect in cystic fibrosis model ferrets
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Kate JDA Excoffon, PhD (she/her/hers) – Spirovant Sciences, Inc
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.4- Prime editing functionally corrects rare and common cystic-fibrosis causing mutations in patient-derived organoids and airway epithelia
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Mattijs Bulcaen, MA (he/him/his) – KU Leuven
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.5- Functional exploration of codon optimization in CFTR
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Jeffrey T. Gabell, M.S. (he/him/his) – University of Rochester School of Medicine and Dentistry
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.6- Examining variation in Intron 1 of CFTR relative to self-reported and genomic defined race
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Guntaas S. Shergill, HBSc – Washington University in St. Louis
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.7- A CFTR endless RNA (eRNA) restores function with increased and prolonged activity and expression in 16HBEs
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Sunny Xia, PhD – Pioneering Medicines
    PTAC/CFTR
  • Thursday, Sep 26th
    9:00 AM – 9:50 AM ET
    RFPT01.8- RCT2100 rescue of CFTR function in CF patient human bronchial epithelial cells and mucociliary clearance in CF ferrets
    Location: Theater B (Exhibit & Poster Hall)
    Speaker: Marco Weinberg, PhD (he/him/his) – Recode Therapeutics
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W03--New Insights Regarding CFTR Basic Biology & Implications for Future Therapeutics
    Location: 157 A-C
    Chair: Carlos M. Farinha, PhD (he/him/his) – Faculty of Sciences, University of Lisboa, BioISI - Biosystems and Integrative Sciences Institute
    Chair: Kenichi Okuda – Marsico Lung Institute/Cystic Fibrosis Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W05--Advances Along the Path to a Cure
    Location: 210 A-C
    Chair: John D. Lueck, Ph.D. – University of Rochester School of Medicine and Dentistry
    Chair: Alexandra S. Piotrowski-Daspit, PhD (she/her/hers) – University of Michigan
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W03.1- Allosteric inhibition of CFTR gating by CFTRinh-172 binding in the pore
    Location: 157 A-C
    Speaker: Xiaolong Gao, PhD – University of Missouri-Columbia
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W03.2- Enhancing mutant CFTR expression: insights into Extracellular Loop functionality
    Location: 157 A-C
    Speaker: Yunjie Huang, PhD – Indiana University
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W03.3- CFTR-F508del is targeted for endolysosomal degradation in the presence of small-molecule correctors
    Location: 157 A-C
    Speaker: Celeste Riepe, PhD (she/her/hers) – Stanford University
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W03.4- Pulmonary Ionocytes Regulate Chloride Secretion and Absorption via CFTR
    Location: 157 A-C
    Speaker: Feng Yuan, PhD – University of Iowa
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W03.5- BEST4+ CFTR High Expresser cells in normal rat are neuropods that sense and respond to luminal pH and are altered in dF508 CF intestine
    Location: 157 A-C
    Presenting Author: Nadia A. Ameen, MD (she/her/hers) – Yale School Medicine
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    In vitro delivery of CFTR gene cargo using DNA nanocarriers
    Location: 210 A-C
    Speaker: Isabella Ferranti (she/her/hers) – Carnegie Mellon University
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    Optimized CFTR E2-27 super exon cargos rescue CFTR function
    Location: 210 A-C
    Speaker: Kezhi Yan, PhD – Cystic Fibrosis Foundation
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    Splice-switching antisense oligonucleotides to recover CFTR function disrupted by C-terminal truncating nucleotide variants
    Location: 210 A-C
    Speaker: Wren Michaels, PhD (she/her/hers) – Rosalind Franklin University of Medicine and Science
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W05.4- AAV1 or 6-Δ27-264 CFTR successfully ameliorated clinical symptoms in G551D Ferrets.
    Location: 210 A-C
    Speaker: Liudmila Cebotaru, MD (she/her/hers) – Johns Hopkins University
    PTAC/CFTR
  • Thursday, Sep 26th
    10:15 AM – 12:15 PM ET
    W05.5- Poly(amine-co-ester) (PACE) nanoparticle delivery of CFTR mRNA shows restoration of CFTR activity in cystic fibrosis airway models.
    Location: 210 A-C
    Speaker: Ju Hyun Lee, MM, MPhil, MS, MPhil – Yale University
    PTAC/CFTR
  • Thursday, Sep 26th
    12:30 PM – 1:20 PM ET
    FSC01--Fireside Chat Phil Thomas
    Location: Fireside Chat A (Exhibit & Poster Hall)
    Speaker: Phil Thomas, PhD (he/him/his) – UT Southwestern
    PTAC/CFTR
  • Thursday, Sep 26th
    12:30 PM – 2:00 PM ET
    LL02--Alveoli: A Player in the CF Lung?
    Location: 259 AB
    Chair: Wanda O'Neal – University of North Carolina at Chapel Hill Departments of Medicine and Genetics
    Chair: Raymond J. Pickles, PhD – University of North Carolina at Chapel Hill
    Speaker: Raymond J. Pickles, PhD – University of North Carolina at Chapel Hill
    Speaker: Wanda O'Neal – University of North Carolina at Chapel Hill Departments of Medicine and Genetics
    APPTTPTAC/CFTR
  • Thursday, Sep 26th
    2:30 PM – 4:30 PM ET
    S07--CFTR 2024: Better Understanding of the Underlying CF Disease Mechanism
    Location: 104 C
    Chair: Jeff L. Brodsky, PhD (he/him/his) – University of Pittsburgh
    Chair: Kathryn E. Oliver, PhD, MS (she/her/hers) – Emory University School of Medicine
    PTAC/CFTR
  • Thursday, Sep 26th
    2:30 PM – 4:30 PM ET
    S11--Nucleotide-Based Therapeutics Across Diverse Populations
    Location: 157 A-C
    Chair: Jennifer L. Taylor-Cousar, MD (she/her/hers) – National Jewish Health
    Chair: Nicole Mayer Hamblett, PhD (she/her/hers) – University of Washington
    PTAC/CFTR
  • Friday, Sep 27th
    7:45 AM – 9:45 AM ET
    W16--From the Gene Editor's Desk: Rewriting Mutant CFTR Using CRISPR & Other Technologies
    Location: 157 A-C
    Chair: Sriram Vaidyanathan – Nationwide Children’s Hospital, The Ohio State University, Columbus, OH, 43215
    Chair: Greg Newby – Johns Hopkins University School of Medicine
    PTAC/CFTR
  • Friday, Sep 27th
    7:45 AM – 9:45 AM ET
    W16.1- Internal chemical modifications in ssDNA templates enable improved CFTR gene correction and primary airway cell modeling compared to end-modified ssDNA templates
    Location: 157 A-C
    Speaker: Karen L. Kanke (she/her/hers) – Nationwide Children's Hospital
    PTAC/CFTR
  • Friday, Sep 27th
    7:45 AM – 9:45 AM ET
    W16.2- Virus-like particle mediated Cas9/gRNA delivery with a CFTR super exon rescues CFTR function in differentiated primary human bronchial epithelial cells
    Location: 157 A-C
    Speaker: Ariel Aspiras, PhD – Cystic Fibrosis Foundation
    PTAC/CFTR
  • Friday, Sep 27th
    7:45 AM – 9:45 AM ET
    W16.3- Polymeric Nanoparticles for In Utero Gene Delivery in Non-Human Primates
    Location: 157 A-C
    Speaker: Alexandra S. Piotrowski-Daspit, PhD (she/her/hers) – University of Michigan
    PTAC/CFTR
  • Friday, Sep 27th
    7:45 AM – 9:45 AM ET
    W16.4- Rescuing G542X by Adenine Base Editing: A guide to restore function
    Location: 157 A-C
    Speaker: Lucia Nicosia, MSc – University College Cork
    PTAC/CFTR
  • Friday, Sep 27th
    7:45 AM – 9:45 AM ET
    W16.5- Prime editing enables efficient, precise correction of CFTR F508del and functional rescue in human airway epithelial cells
    Location: 157 A-C
    Speaker: Colin Hemez (he/him/his) – Broad Institute
    PTAC/CFTR
  • Friday, Sep 27th
    10:15 AM – 12:15 PM ET
    W20--Model Systems to Study Non-pulmonary Manifestations of CF
    Location: 104 C
    Chair: Ben Ross
    Chair: Kyu Shik Mun, PhD – Cedars-Sinai Medical Center
    EPPIMIPTAC/CFTR
  • Friday, Sep 27th
    10:15 AM – 12:15 PM ET
    W20.1- CF mouse model reveals novel roles of CFTR in the kidney and demonstrates features of progressive kidney disease.
    Location: 104 C
    Speaker: Agnieszka Swiatecka-Urban, MD – University of Virginia
    EPPIMIPTAC/CFTR
  • Friday, Sep 27th
    10:15 AM – 12:15 PM ET
    W20.2- Recovery of pancreatic functions in CFTR-G551D ferrets after ivacaftor
    Location: 104 C
    Speaker: Yaling Yi, MS – University of Iowa
    EPPIMIPTAC/CFTR
  • Friday, Sep 27th
    10:15 AM – 12:15 PM ET
    W20.3- Lactic acid producing bacteria are increased in CFTR-deficient colon crypt microbiota and lactate potentiates clonogenicity and stem cell capacity in colonic organoids and cancer cells.
    Location: 104 C
    Speaker: Patricia Scott, PhD – Univ. of MN Medical School
    EPPIMIPTAC/CFTR
  • Friday, Sep 27th
    10:15 AM – 12:15 PM ET
    W20.4- The role of micropeptide BNLN in Cystic Fibrosis Related Diabetes
    Location: 104 C
    Speaker: Tate Neff – University of Iowa
    EPPIMIPTAC/CFTR
  • Friday, Sep 27th
    10:15 AM – 12:15 PM ET
    W20.5- Co-culture of mouse enteroids and sensory neurons as a model system to study the gut-brain axis in CF
    Location: 104 C
    Speaker: Oscar O. Morales, MSc (he/him/his) – University of Saskatchewan
    EPPIMIPTAC/CFTR
  • Friday, Sep 27th
    2:30 PM – 4:30 PM ET
    S15--Challenges to Efficient, Effective, & Durable CFTR Rescue
    Location: 157 A-C
    Chair: Martin Mense – CFFT Lab, Cystic Fibrosis Foundation
    Chair: Marianne Carlon – KU Leuven
    PTAC/CFTR
  • Friday, Sep 27th
    2:30 PM – 4:30 PM ET
    S22--Finding an “Optimal” Dose in Drug Development: The Importance of Exposure/Response Relationships for Advancing Chronic CF Therapies
    Location: 210 A-C
    Chair: Donald R. VanDevanter, PhD – Case Western Reserve University School of Medicine
    Chair: Nicole Mayer Hamblett, PhD (she/her/hers) – University of Washington
    APPTTCLINPHARMPTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W31--Progress Overcoming Premature Stop Codons in CFTR
    Location: 157 A-C
    Chair: David M. Bedwell – Departments of Biochemistry & Molecular Genetics, University of Alabama at Birmingham (UAB), Birmingham, AL
    Chair: Michelle L. Hastings, PhD (she/her/hers) – University of Michigan
    PTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W32--Airway Differentiation, Pathogenesis, & Repair
    Location: 153 A-C
    Chair: Margarida Amaral – FCUL-Faculty of Sciences, University of Lisboa
    Chair: Vibha Lama, MD MS – Emory University, Atlanta, GA
    Chair: Gianni Carraro, PhD – Cedars-Sinai Medical Center
    APPTTPTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W31.1- Pharmacological rescue of CFTR premature termination codon (PTC) variants
    Location: 157 A-C
    Speaker: Emily Bulik-Sullivan, n/a (she/her/hers) – UNC-Chapel Hill
    PTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W31.2- CFTR PTC readthrough can be enhanced by a transcript-specific ASO approach
    Location: 157 A-C
    Speaker: Jae seok Yoon, PhD (he/him/his) – Cystic Fibrosis Foundation
    PTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W31.3- Rescue of CFTR nonsense mutations is enhanced under inflammatory stimuli
    Location: 157 A-C
    Speaker: Anna Borrelli, N/A (she/her/hers) – TIGEM- Telethon Institute of Genetics and Medicine
    PTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W31.4- Determining the functional consequences of translational readthrough at common CFTR nonsense mutations
    Location: 157 A-C
    Speaker: Kari Thrasher, PhD – University of Alabama at Birmingham
    PTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W31.5- Global suppression of nonsense-mediated mRNA decay exacerbates the inflammatory response
    Location: 157 A-C
    Speaker: Zhongyou Li, PhD (he/him/his) – University of Colorado Anschutz Medical Campus
    PTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W32.1- Lef1 Induces Ionocyte Specification of Submucosal Gland Progenitors. Which One?
    Location: 153 A-C
    Speaker: Weam S A Shahin, MD PhD – University of Iowa
    APPTTPTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W32.2- Ionocytes localized in airway submucosal gland mucus tubule and ciliated ducts contribute to mucin unpacking
    Location: 153 A-C
    Speaker: Amirala Ostad Mohammad Nazari, BSc (he/him/his) – Department of Anatomy, Physiology and Pharmacology, University of Saskatchewan, Saskatoon, SK, Canada
    APPTTPTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W32.3- Temporal Dynamics in the Regulation of Pulmonary Ionocyte Development through NOTCH Signaling
    Location: 153 A-C
    APPTTPTAC/CFTR
  • Saturday, Sep 28th
    7:45 AM – 9:45 AM ET
    W32.4- ELD607, an Orai1 antagonist, reverses goblet cell metaplasia in βENaC mice.
    Location: 153 A-C
    Speaker: Saira Ahmad, PhD – Eldec Pharmaceuticals
    APPTTPTAC/CFTR
  • Saturday, Sep 28th
    10:15 AM – 12:15 PM ET
    S27--Airway Stem Cells: Tools, Targets, & Therapies
    Location: 258 A-C
    Chair: Viral Shah – MGH
    Chair: Amy L. Ryan, PhD – University of Iowa
    PTAC/CFTR
  • Saturday, Sep 28th
    12:30 PM – 2:00 PM ET
    RT1-PTAC/CFTR: Theratyping CFTR Variants
    Location: 208
    Facilitator: Neeraj Sharma, PhD, DVM – Johns Hopkins University School of Medicine
    PTAC/CFTR
  • Saturday, Sep 28th
    12:30 PM – 2:00 PM ET
    RT2-PTAC/CFTR: The Structural Biology of CFTR
    Location: 208
    Facilitator: Han-I Yeh, PhD – University of Missouri
    PTAC/CFTR
  • Saturday, Sep 28th
    12:30 PM – 2:00 PM ET
    RT3-PTAC/CFTR: Illuminating the Impact of CFTR in Cancer
    Location: 208
    PTAC/CFTR
  • Saturday, Sep 28th
    12:30 PM – 2:00 PM ET
    RT4-PTAC/CFTR: Models & Assays for CFTR Research
    Location: 208
    Facilitator: Deborah M. Cholon, PhD – Marsico Lung Institute/Cystic Fibrosis Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
    PTAC/CFTR
  • Saturday, Sep 28th
    12:30 PM – 2:00 PM ET
    RT5-PTAC/CFTR: Illuminating the Impact of CFTR in Development
    Location: 208
    Facilitator: Amy P. Wong, PhD (she/her/hers) – The Hospital for Sick Children
    PTAC/CFTR
  • Saturday, Sep 28th
    12:30 PM – 2:00 PM ET
    RT6-PTAC/CFTR: CFTR Outside the Respiratory Track: Importance to Fully Understanding the Disease
    Location: 208
    Facilitator: Wanda O'Neal – University of North Carolina at Chapel Hill Departments of Medicine and Genetics
    PTAC/CFTR