The Team & Mission

MISSION STATEMENT

Our vision is to establish a research program for childhood lung disease that brings investigators from diverse research backgrounds in a focused effort to study and develop new and innovative therapies. The mission of this endeavor will entail 3 focus areas tailored for discovery and treatment of lung disease and help build one of the first comprehensive research programs for rare lung disease in children.

Disease Modeling
Disease modeling is composed of two objectives: one, continued registration for a biorepository of tissue to gain knowledge of the disease process and two, the establishment of animal models of human lung disease. CHOP is one of the largest referral centers in the United States for pediatric lung disease, and Dr. David Frank has initiated a pediatric lung tissue repository at CHOP in conjunction with adult tissue collection for the Penn Center for Pulmonary Biology.  

Cellular and Target Discovery
In order to begin to treat disease, an understanding of the normal process of how the lung is first built and also undergoes regeneration after injury is essential. This focus will help identify important lung progenitor cells, stem cells, and the supporting cells that influence growth and regeneration that could be targeted for therapy.

Genetics, Gene Editing, and Targeted Therapy
Novel therapies are the ultimate mission of this endeavor and include the continued discovery of genetic anomalies in childhood lung airway and vascular disease, advancement of recently developed technologies at CHOP and Penn for in utero gene editing and correction of gene mutations postnatally, and the selective delivery of therapies to the lungs to treat these diseases

The short-term goals of this collaborative effort will be to increase clinical research coordinated recruitment and collection of all available pediatric lung tissue at CHOP that will be available to investigators all over the world, acquire the ability to develop high output of novel human disease-based animal models for novel gene, drug, and cellular therapies, and the continued recruitment of additional investigators focused on childhood lung disease.

We believe that the initial success in meeting our short-term goals will help accomplish the mission and long-term goals of this program that include:

• Largest lung tissue biorepository that will provide samples to the scientific community throughout the world

• Multidisciplinary Center for Pediatric Lung Research composed of a collaborative effort of CHOP and Penn investigators

• Establishment of targeted cellular, drug, and gene therapies toward lung vascular abnormalities

Selected Publications