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publicationDate 2019-07-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2019201551-A1
titleOfInvention Pulmonary targeted cas9/crispr for in vivo editing of disease genes
abstract An adenovirus vector comprising: a pulmonary targeting coding sequence, CRISPR components such as a Cas9 coding sequence, and a guideRNA coding sequence which can be used for gene therapy. The adenovirus can be a gorilla adenovirus, and can include a pulmonary cell targeting sequence such as an MBP targeting ligand coding sequence. The adenovirus can be targeted to pulmonary epithelium with a vascular specific promoter and integrin targeting peptides incorporated into a viral knob. The adenovirus can be used to introduce serum proteins via the pulmonary epithelium. Hemophilia can be treated by adenoviral introduction of factor VIII or factor IX.
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