{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T23:51:08Z","timestamp":1793317868473,"version":"4.5.0"},"reference-count":101,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2023,6,30]],"date-time":"2023-06-30T00:00:00Z","timestamp":1688083200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004281","name":"Narodowe Centrum Nauki","doi-asserted-by":"publisher","award":["UMO-2020\/37\/N\/NZ2\/01125"],"award-info":[{"award-number":["UMO-2020\/37\/N\/NZ2\/01125"]}],"id":[{"id":"10.13039\/501100004281","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Bioeng. Biotechnol."],"abstract":"<jats:p><jats:bold>Introduction:<\/jats:bold> The benefits of patient\u2019s specific cell\/gene therapy have been reported in relation to numerous genetic related disorders including osteogenesis imperfecta (OI). In osteogenesis imperfecta particularly also a drug therapy based on the administration of bisphosphonates partially helped to ease the symptoms.<\/jats:p><jats:p><jats:bold>Methods:<\/jats:bold> In this controlled trial, fibroblasts derived from patient diagnosed with OI type II have been successfully reprogrammed into induced Pluripotent Stem cells (iPSCs) using Yamanaka factors. Those cells were subjected to repair mutations found in the <jats:italic>COL1A1<\/jats:italic> gene using homologous recombination (HR) approach facilitated with star polymer (STAR) as a carrier of the genetic material.<\/jats:p><jats:p><jats:bold>Results:<\/jats:bold> Delivery of the correct linear DNA fragment to the osteogenesis imperfecta patient\u2019s cells resulted in the repair of the DNA mutation with an 84% success rate. IPSCs showed 87% viability after STAR treatment and 82% with its polyplex.<\/jats:p><jats:p><jats:bold>Discussion:<\/jats:bold> The use of novel polymer Poly[N,N-Dimethylaminoethyl Methacrylate-co-Hydroxyl-Bearing Oligo(Ethylene Glycol) Methacrylate] Arms (P(DMAEMA-co-OEGMA-OH) with star-like structure has been shown as an efficient tool for nucleic acids delivery into cells (Funded by National Science Centre, Contract No. UMO-2020\/37\/N\/NZ2\/01125).<\/jats:p>","DOI":"10.3389\/fbioe.2023.1205122","type":"journal-article","created":{"date-parts":[[2023,6,30]],"date-time":"2023-06-30T18:39:06Z","timestamp":1688150346000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":13,"title":["Gene-repaired iPS cells as novel approach for patient with osteogenesis imperfecta"],"prefix":"10.3389","volume":"11","author":[{"given":"Agnieszka","family":"Fus-Kujawa","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Mendrek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karolina","family":"Bajdak-Rusinek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalia","family":"Diak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karolina","family":"Strzelec","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ewa","family":"Gutmajster","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kamil","family":"Janelt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Agnieszka","family":"Kowalczuk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Trybus","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrycja","family":"Rozwadowska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wojciech","family":"Wojakowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katarzyna","family":"Gawron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aleksander L.","family":"Siero\u0144","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2023,6,30]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"e20674","DOI":"10.7554\/eLife.20674","article-title":"Addressing the ethical issues raised by synthetic human entities with embryo-like features","volume":"6","author":"Aach","year":"2017","journal-title":"eLife"},{"key":"B2","doi-asserted-by":"publisher","first-page":"3213","DOI":"10.1021\/bm2006906","article-title":"Cationic amphiphilic star and linear block copolymers: Synthesis, self-assembly, and in vitro gene transfection","volume":"12","author":"Alhoranta","year":"2011","journal-title":"Biomacromolecules"},{"key":"B3","doi-asserted-by":"publisher","first-page":"663","DOI":"10.3390\/cancers12030663","article-title":"Cellular functions of OCT-3\/4 regulated by ubiquitination in proliferating cells","volume":"12","author":"Baek","year":"2020","journal-title":"Cancers"},{"key":"B4","doi-asserted-by":"publisher","first-page":"4324","DOI":"10.1016\/j.actbio.2012.07.034","article-title":"PDMS(star)-PEG hydrogels prepared via solvent-induced phase separation (SIPS) and their potential utility as tissue engineering scaffolds","volume":"8","author":"Bailey","year":"2012","journal-title":"Acta Biomater."},{"key":"B5","doi-asserted-by":"publisher","first-page":"3483","DOI":"10.3390\/cells10123483","article-title":"Human induced pluripotent stem cell as a disease modeling and drug development platform - a cardiac perspective","volume":"10","author":"Bekhite","year":"2021","journal-title":"Cells"},{"key":"B6","doi-asserted-by":"publisher","first-page":"100","DOI":"10.1242\/bio.022111","article-title":"Systematic evaluation of markers used for the identification of human induced pluripotent stem cells","volume":"6","author":"Bharathan","year":"2017","journal-title":"Biol. 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