Final Program - American Society of Gene & Cell Therapy

Program Schedule, Wednesday, May 15, 2013
Wednesday, May 15, 2013
Scientific Symposium 104
8:00 am - 10:00 am
ROOM: 151 ABCG
Novel Structural and Functional Assessment of the Respiratory Tract: Potential New Endpoints
for Gene and Cell Therapy
CO-CHAIRS: David A. Dean, PhD and Christian Mueller, PhD
SPEAKERS
David W. Parsons, PhD
Developments in Functional X-ray Imaging: ASL, MCT and “Pinpoint Spirometry”
Driven by a desire to rapidly, accurately and reliably measure the effect of airway gene therapy treatments for Cystic Fibrosis
in mice, we have been developing several novel analytical options for airways using synchrotron phase contrast X-ray. The high
lux and coherence of synchrotron X-rays underlie our ability to visualize mouse airway-surface anatomy and activity in-vivo, to
sub-micron resolution; ultra-fast imaging of dynamic lung movement in 4-dimensions to reveal regional airlows is also enabled.
Importantly, all of these new approaches are designed to be non-invasive and allow airway health and function in intact animals
to be examined. Alterations in the depth of the airway surface liquid (ASL) and altered Mucociliary Transit (MCT) are the earliest
physiological effects of CFTR activity that can produce visible change in airway surface structure and function, and are key markers
of CF airway health to be improved by an effective gene therapy. The core methods for the image capture, analysis, and presentation
of both ASL and MCT markers are rapidly developing to provide new endpoint tools. The application of velocimetry techniques to
synchrotron CT data has created a powerful new tool for regional lung health assessment (”pinpoint spirometry”) that is likely to be
of particular value in animal models, and is also well suited to translation into a clinically-applicable system.
Cecilia Lo, PhD
Respiratory Motile Cilia Dysfunction in Congenital Heart Disease Patients and Contribution to Worse
Postsurgical Outcomes
Steven M. Rowe, MD
Functional Anatomic Imaging of the CF Airway
A critical barrier causing a roadblock in our understanding of CF pathogenesis is the lack of tools available for visualizing the
important microstructural, functional, and biomechanical features of the respiratory mucosa and mucus in vivo. Recently, our
laboratories have developed an innovative technology, termed 1-µm resolution optical coherence tomography (µOCT), which
enables real-time cross-sectional microscopy of the functional epithelial surface of living airways and can be readily combined with
measures of ion transport in vivo. With µOCT, we have been able to simultaneously and quantitatively monitor airway surface liquid
(ASL) and periciliary layer (PCL) depths, ciliary beating, and mucociliary transport (MCT) in situ, while also measuring mucus
viscosity by native particle tracking techniques without the use of contrast dyes, exogenous microparticles, or other manipulations.
Key manifestations of the underlying CF defect can readily be observed, and an in vivo probe is currently under development. In
addition to answering important questions regarding CF pathophysiology, this technology provides a clear path towards the use of
µOCT for monitoring functional responses to novel therapeutics.
Maria P. Limberis, PhD
Development of an AAV-based Prophylactic Vaccine against Pandemic Inluenza
We will discuss the development of an effective prophylactic vaccine against inluenza. This vaccine is based on AAV9 vector and
expresses a broadly neutralizing antibody against inluenza that protects relevant animal models against challenge with H5N1 and
H1N1.
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013