Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes
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Cellular and Molecular Life Sciences
REVIEW
Pluripotent stem cell‑based gene therapy approach: human de novo synthesized chromosomes Sergey A. Sinenko1 · Sergey V. Ponomartsev1 · Alexey N. Tomilin1,2 Received: 25 May 2020 / Revised: 14 September 2020 / Accepted: 22 September 2020 © Springer Nature Switzerland AG 2020
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. Keyword Alphoid HAC · Stem cell-based therapy · Induced pluripotent stem cells (iPSCs) · Embryonic stem cells (ESCs) · Microcell-mediated chromosome transfer (MMCT) · Gene loading vectors Abbreviations BACs Bacterial artificial chromosomes CENP-B Centromere protein B cGMP Current good manufacturing practice GFP Green fluorescent protein CHO Chinese hamster ovary ESCs Embryonic stem cells IIS Iterative integration system iMCT Isolated metaphase chromosome transfection iPSCs Induced pluripotent stem cells FVIII Human clotting factor VIII HAC Human artificial chromosome hESCs Human embryonic stem cells hiPSCs Human induced pluripotent stem cells
* Sergey A. Sinenko [email protected] * Alexey N. Tomilin [email protected] 1
Institute of Cytology, Russian Academy of Sciences, 4 Tikhoretsky Ave, St‑Petersburg 194064, Russia
Institute of Translational Biomedicine, St-Petersburg State University, 7–9, Universitetskaya Emb, St‑Petersburg 199034, Russia
2
HPRT Hypoxanthine–guanine phosphoribosyl transferase HSV-1 Herpes simplex virus 1 HSPCs Hematopoietic stem and progenitor cells HVJ-E Hemagglutinating virus of Japan E lacO Lac operon loxP Locus of X-over P1 MLV Murine leukemia retrovirus MMCT Microcell mediated chromosome transfer MSCs Mesenchymal stem cells MV Measles virus PEG Polyethylene glycol PSCs Pluripotent stem cells rAAVs Adeno-associated recombinant viruses RMCE Recombinase-mediated cassette exchange method TAR Transformation associated recombination tetO Tetracycline operator tetR Tet-repressor protein SIM Sequential integration of multiple vectors YACs Yeast artificial chromosomes
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Introduction Gene therapy represents DNA-manipula
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