Invention:
This technology describes nanoparticles with specific structure design and modification for high efficiency and specific delivery of nucleic acids to the pulmonary endothelial cells. After intravenous administration, the nanoparticles were capable of delivering non-integrating DNA plasmids to lung microvascular endothelial cells but not to other lung cell types. Hematologic analysis and liver and kidney metabolic panels demonstrated the lack of toxicity in experimental mice. Thus, these nanoparticles can be an ideal vehicle for gene therapy targeting lung microvascular endothelium in pulmonary vascular disorders.
Background:
Lung endothelial cell dysfunction are primarily responsible for a variety of acute and chronic pulmonary diseases including pulmonary hypertension, viral and bacterial pneumonia, bronchopulmonary dysplasia, and congenital lung diseases such as alveolar capillary dysplasia with misalignment of pulmonary veins. While several nanoparticle delivery systems targeting endothelial cells have been recently developed, none of them is specific to lung endothelial cells without targeting other organs in the body.
Applications:
- Lung epithelial cell disorders
- Targeted genetic therapy
- Precision platform for vascular diseases gene therapy
- Cancer
- Research and clinical uses
Advantages:
- Previously proven non-toxicity in a mouse model
- Paves the way for targeted lung therapies
- First of its kind (non-toxic nanoparticle delivery system)
