title: Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies.
abstract: BACKGROUND
  The immunogenicity and limited stability of conventional messenger RNA (mRNA) has traditionally restricted its potential therapeutic use. In 1992, the first clinical application of mRNA was reported as a potential protein-replacement therapy; however, subsequent investigations have not been made for almost two decades. Recent developments, including increased stability, controlling immunogenicity, as well as utilization of mRNA encoding zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR-Cas9, have implicated modified mRNA as a very promising option for cancer immunotherapy, vaccines, protein expression replacement, and genome editing. This review aims to offer a summary of our present understanding of and improvements in mRNA-based drug technologies, along with a focus on the role in therapeutic options for pediatric respiratory diseases and hemoglobinopathies.
  
  
  CONCLUSIONS
  This mini review summarizes the recent advances in modified mRNA-based therapy and its potential therapeutic effect in treating major pediatric diseases.
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tübingen, Tübingen, Germany.
authors.forename: Justin S
authors.initials: JS
authors.lastname: Antony
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tübingen, Tübingen, Germany.
authors.forename: Alexander
authors.initials: A
authors.lastname: Dewerth
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tübingen, Tübingen, Germany.
authors.forename: Ashiqul
authors.initials: A
authors.lastname: Haque
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tübingen, Tübingen, Germany.
authors.forename: Rupert
authors.initials: R
authors.lastname: Handgretinger
authors.affiliation: Department of Pediatrics I-Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tübingen, Tübingen, Germany. michael.kormann@med.uni-tuebingen.de.
authors.forename: Michael S D
authors.initials: MS
authors.lastname: Kormann
keywords: Gene therapy; Modified mRNA; Pediatric diseases