Spliceosome-Mediated RNATrans-Splicing with Recombinant Adeno-Associated Virus Partially Restores Cystic Fibrosis Transmembrane Conductance Regulator Function to Polarized Human Cystic Fibrosis Airway Epithelial Cells

Author:

Liu Xiaoming12,Luo Meihui1,Zhang Liang N.1,Yan Ziying12,Zak Roman1,Ding Wei1,Mansfield S. Gary3,Mitchell Lloyd G.3,Engelhardt John F.12

Affiliation:

1. Department of Anatomy and Department of Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242.

2. Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases, Carver College of Medicine, University of Iowa, Iowa City, IA 52242.

3. Intronn Inc., Gaithersburg, MD 20878.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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3. COL7A1 Editing via RNA Trans-Splicing in RDEB-Derived Skin Equivalents;International Journal of Molecular Sciences;2023-02-22

4. Alternative Splicing and Cancer;Nucleic Acid Biology and its Application in Human Diseases;2023

5. Perspectives on Genetic Medicine for Cystic Fibrosis;Current Gene Therapy;2022-10

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