ABSTRACT Targeted integration of a therapeutic gene at specific loci in safe genomic regions by a non-viral vector can restore the function of the damaged gene. This approach also minimizes

the potential genotoxic effects of transferred DNA. In this study, we have developed a non-viral vector that functions according to site-specific recombination (SSR). The vector contained a

bacterial backbone and puromycin resistance gene (_pur__r_), a β-globin expressing cassette and an _attB_ recombination site. We used phiC31 integrase to insert a copy of the vector into

specific genomic locations of a human hematopoietic cell line. Site-specific integration of the vector with one or two copies in the transcriptionally active regions of the genome was

powerful tool for _ex vivo_ correction of β-globinopathies such as β-thalassemia through successful genomic integration of a functional copy of the globin gene into the patient’s target

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support SIMILAR CONTENT BEING VIEWED BY OTHERS LENTIVIRAL GLOBIN GENE THERAPY WITH REDUCED-INTENSITY CONDITIONING IN ADULTS WITH Β-THALASSEMIA: A PHASE 1 TRIAL Article 03 January 2022 GENE

REPLACEMENT OF Α-GLOBIN WITH Β-GLOBIN RESTORES HEMOGLOBIN BALANCE IN Β-THALASSEMIA-DERIVED HEMATOPOIETIC STEM AND PROGENITOR CELLS Article 18 March 2021 THERAPEUTIC ADENINE BASE EDITING OF

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sincerest gratitude to Professor Michelle Calos for providing the pDB2 and pCMV/Int vectors. This study was funded by grants provided by Royan Institute. AUTHOR INFORMATION AUTHORS AND

AFFILIATIONS * Department of Pharmaceutical Biotechnology and Isfahan Pharmaceutical Sciences Research Center, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical

Sciences, Isfahan, Iran K Dormiani, H Mir Mohammad Sadeghi & H Sadeghi-Aliabadi * Department of Molecular Biotechnology at Cell Science Research Center, Royan Institute for

Biotechnology, ACECR, Isfahan, Iran K Ghaedi, M Forouzanfar & M H Nasr-Esfahani * Biology Department, School of Sciences, University of Isfahan, Isfahan, Iran K Ghaedi * Department of

Stem Cells and Developmental Biology at Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran H Baharvand * Department of Developmental

Biology, University of Science and Culture, ACECR, Tehran, Iran H Baharvand Authors * K Dormiani View author publications You can also search for this author inPubMed Google Scholar * H Mir

Mohammad Sadeghi View author publications You can also search for this author inPubMed Google Scholar * H Sadeghi-Aliabadi View author publications You can also search for this author

inPubMed Google Scholar * K Ghaedi View author publications You can also search for this author inPubMed Google Scholar * M Forouzanfar View author publications You can also search for this

author inPubMed Google Scholar * H Baharvand View author publications You can also search for this author inPubMed Google Scholar * M H Nasr-Esfahani View author publications You can also

search for this author inPubMed Google Scholar CORRESPONDING AUTHORS Correspondence to H Baharvand or M H Nasr-Esfahani. ETHICS DECLARATIONS COMPETING INTERESTS The authors declare no

conflict of interest. ADDITIONAL INFORMATION Supplementary Information accompanies this paper on Gene Therapy website SUPPLEMENTARY INFORMATION SUPPLEMENTARY FIGURE LEGENDS (DOC 26 KB)

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Sadeghi, H., Sadeghi-Aliabadi, H. _et al._ Long-term and efficient expression of human β-globin gene in a hematopoietic cell line using a new site-specific integrating non-viral system.

_Gene Ther_ 22, 663–674 (2015). https://doi.org/10.1038/gt.2015.30 Download citation * Received: 24 September 2014 * Revised: 07 March 2015 * Accepted: 16 March 2015 * Published: 01 April

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