Messenger RNA delivery of a cartilage-anabolic transcription factor as a disease-modifying strategy for osteoarthritis treatment

Sci Rep. 2016 Jan 5:6:18743. doi: 10.1038/srep18743.

Abstract

Osteoarthritis (OA) is a chronic degenerative joint disease and a major health problem in the elderly population. No disease-modifying osteoarthritis drug (DMOAD) has been made available for clinical use. Here we present a disease-modifying strategy for OA, focusing on messenger RNA (mRNA) delivery of a therapeutic transcription factor using polyethylene glycol (PEG)-polyamino acid block copolymer-based polyplex nanomicelles. When polyplex nanomicelles carrying the cartilage-anabolic, runt-related transcription factor (RUNX) 1 mRNA were injected into mouse OA knee joints, OA progression was significantly suppressed compared with the non-treatment control. Expressions of cartilage-anabolic markers and proliferation were augmented in articular chondrocytes of the RUNX1-injected knees. Thus, this study provides a proof of concept of the treatment of degenerative diseases such as OA by the in situ mRNA delivery of therapeutic transcription factors; the presented approach will directly connect basic findings on disease-protective or tissue-regenerating factors to disease treatment.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cartilage, Articular / metabolism*
  • Cartilage, Articular / pathology
  • Disease Models, Animal
  • Gene Expression
  • Gene Transfer Techniques
  • Genes, Reporter
  • Injections, Intra-Articular
  • Mice
  • Micelles
  • Nanocomposites / administration & dosage
  • Nanocomposites / chemistry
  • Osteoarthritis / genetics*
  • Osteoarthritis / metabolism*
  • Osteoarthritis / pathology
  • Osteoarthritis / therapy
  • Osteoarthritis, Knee / genetics
  • Osteoarthritis, Knee / metabolism
  • Osteoarthritis, Knee / pathology
  • Osteoarthritis, Knee / therapy
  • RNA, Messenger / administration & dosage*
  • RNA, Messenger / chemistry
  • RNA, Messenger / genetics*
  • Transcription Factors / genetics*
  • Transcription Factors / metabolism

Substances

  • Micelles
  • RNA, Messenger
  • Transcription Factors