Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes

Sergey A. Sinenko, Sergey V. Ponomartsev, Alexey N. Tomilin

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

Abstract

A novel approach in gene therapy was introduced 20 years ago since artificial non-integrative chromosome-based vectors containing gene loci size inserts were engineered. To date, different human artificial chromosomes (HAC) were generated with the use of de novo construction or “top-down” engineering approaches. The HAC-based therapeutic approach includes ex vivo gene transferring and correction of pluripotent stem cells (PSCs) or highly proliferative modified stem cells. The current progress in the technology of induced PSCs, integrating with the HAC technology, resulted in a novel platform of stem cell-based tissue replacement therapy for the treatment of genetic disease. Nowadays, the sophisticated and laborious HAC technology has significantly improved and is now closer to clinical studies. In here, we reviewed the achievements in the technology of de novo synthesized HACs for a chromosome transfer for developing gene therapy tissue replacement models of monogenic human diseases.

Original languageEnglish
JournalCellular and Molecular Life Sciences
DOIs
StateE-pub ahead of print - 3 Oct 2020

Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Pharmacology
  • Cellular and Molecular Neuroscience
  • Cell Biology

Keywords

  • Alphoid HAC
  • Embryonic stem cells (ESCs)
  • Gene loading vectors
  • Induced pluripotent stem cells (iPSCs)
  • Microcell-mediated chromosome transfer (MMCT)
  • Stem cell-based therapy

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