Lessons learned from lung and liver in-vivo gene therapy: implications for the future.

ABSTRACT:

Introduction

Ex-vivo gene therapy has had significant clinical impact over the last couple of years and in-vivo gene therapy products are being approved for clinical use. Gene therapy and gene editing approaches have huge potential to treat genetic disease and chronic illness.

Areas covered

This article provides a review of in-vivo approaches for gene therapy in the lung and liver, exploiting non-viral and viral vectors with varying serotypes and pseudotypes to target-specific cells. Antibody responses inhibiting viral vectors continue to constrain effective repeat administration. Lessons learned from ex-vivo gene therapy and genome editing are also discussed.

Expert opinion

The fields of lung and liver in-vivo gene therapy are thriving and a comparison highlights obs

SUBMITTER: van Haasteren J 

PROVIDER: S-EPMC6134476 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature