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Project: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases

PI:   Guang-Ping Gao, PhD NIH Report

Efficacy and safety limitations in current gene editing technologies have hindered efforts to treat genetic lung diseases. This proposal seeks to develop and validate a combinatorial delivery approach that uses non-viral and viral vehicles to efficiently transport gene editing tools to disease-relevant cells in the lung. Completion of our work will establish safe and effective delivery vehicles that will guide the design of future gene therapies for genetic disorders.
Summary of data submissions:
  • Data for 2  experiments were submitted on 2020-10-20 SCGE ID:1005
  • Data for 1  experiments were submitted on 2021-09-21 SCGE ID:1051
  • 1 of 3 experiments have been validated

Submissions Details

SCGE ID:1005 - Submission Date: 2020-10-20 
Experiment Name Type Description
Testing AAV5 for activation of tdTomato in HEK293T cells

In Vitro AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T cells by transient transfection. Both delivery and gene editing were detected by fluorescence.
Testing AAV5 for activation of tdTomato in mouse airway

In Vivo AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Viral delivery was detected by GFP expression and gene editing quantified by tdTomato activation
SCGE ID:1051 - Submission Date: 2021-09-21 
Experiment Name Type Description
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells

In Vivo AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Gene editing quantified by tdTomato activation and cell specific markers for club and ciliated cell types.