Litcius/Paper detail

Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy

Laura Marquez‐Loza, Ashley L. Cooney, Qian Dong, Christoph O. Randak, Stefano Rivella, Patrick L. Sinn, Paul B. McCray

2021Molecular Therapy — Methods & Clinical Development26 citationsDOIOpen Access PDF

Abstract

sequences advance lentiviral vectors toward CF gene therapy clinical trials.

Topics & Concepts

Cystic fibrosisGenetic enhancementViral vectorVector (molecular biology)Function (biology)Gene expressionCystic fibrosis transmembrane conductance regulatorComputational biologyBiologyGeneVirologyMedicineCancer researchGeneticsRecombinant DNAVirus-based gene therapy researchEnergy Harvesting in Wireless NetworksCystic Fibrosis Research Advances