{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T20:04:09Z","timestamp":1790021049703,"version":"4.0.1"},"reference-count":0,"publisher":"Bentham Science Publishers Ltd.","content-domain":{"domain":["eurekaselect.com"],"crossmark-restriction":true},"short-container-title":["CGT"],"published-print":{"date-parts":[[2026,3,25]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract:<\/jats:title>\n                    <jats:p>Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the CFTR\ngene, leading to dysfunctional chloride ion transport across epithelial cells. This results in the accumulation\nof thick, sticky mucus, primarily affecting the lungs, pancreas, and gastrointestinal tract,\nleading to chronic respiratory infections and progressive lung damage. Although CFTR modulators\nhave significantly improved outcomes for some patients, their efficacy is limited to specific mutations,\nleaving many without adequate treatment. CFTR-mRNA-based gene therapy serves as an advantageous\ntherapeutic method for all mutations, independent of the treatment target. Through synthetic\nCFTR mRNA delivery to epithelial cells using this method, the cells can synthesize the functional\nCFTR protein required for proper ion transport and mucus hydration. mRNA therapy avoids\nnuclear entry and genomic integration, reducing the risk of insertional mutagenesis and long-term\ngenetic alterations. Lipid nanoparticles, together with other non-viral delivery carriers, demonstrate\nsignificant potential to protect mRNA structures and enhance their ability to enter cells while promoting\nprotein production. Research conducted using animal models has revealed that mRNA therapy\nleads to the successful restoration of CFTR function, accompanied by improved airway hydration\noutcomes. Ongoing challenges exist for this therapy, as mRNA encounters instability issues\nwhile triggering immune reactions and requires constant dose delivery. Multiple scientific research\nfields are collaborating on mRNA engineering advancements, along with the development of delivery\ntechnologies and combination therapy strategies, to overcome existing obstacles. The study examines\ncurrent therapeutic approaches while assessing delivery pathways and pharmaceutical advancements,\nas well as expected research directions regarding CFTR-mRNA therapy, which\ndemonstrates potential to treat all Cystic fibrosis patients through a single, comprehensive treatment.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.2174\/0115665232413980251103133741","type":"journal-article","created":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T18:32:09Z","timestamp":1774809129000},"update-policy":"https:\/\/doi.org\/10.2174\/bsp_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["CFTR mRNA-Based Gene Therapy for Cystic Fibrosis: A Mutation-Agnostic Strategy to Restore Ion Transport Function"],"prefix":"10.2174","volume":"26","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6651-6371","authenticated-orcid":true,"given":"Jayendra","family":"Kumar","sequence":"first","affiliation":[{"name":"Department of Pharmaceutical Chemistry, SRM Modinagar College of Pharmacy, SRM Institute of Science and Technology,Delhi-NCR Campus, Modinagar, Ghaziabad, UP \u2013 201204, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-0605-8569","authenticated-orcid":true,"given":"Mehetre Dattatraya","family":"Martand","sequence":"additional","affiliation":[{"name":"Department of Nephrology, Holy Family Hospital, Okhla Road, New Delhi, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9392-0978","authenticated-orcid":true,"given":"Arpan Kumar","family":"Tripathi","sequence":"additional","affiliation":[{"name":"Faculty of Pharmaceutical Sciences, Shri Shankaraacharya Technical Campus, Bhilai Chhattisgarh, 490001, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0615-1144","authenticated-orcid":true,"given":"Prabhat","family":"Agarwal","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Practice, SGT College of Pharmacy, SGT University, Gurgaon, Haryana, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9611-3127","authenticated-orcid":true,"given":"Hitesh","family":"Kumar","sequence":"additional","affiliation":[{"name":"School of Pharmaceutical Sciences, Om Sterling Global University, Hisar, Haryana, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0005-6103-1243","authenticated-orcid":true,"given":"Shamim","family":"Shamim","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, IIMT College of Medical Sciences, IIMT University, Ganga Nagar (250001), Meerut, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-6832-0907","authenticated-orcid":true,"given":"Shadab","family":"Ali","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, IIMT College of Medical Sciences, IIMT University, Ganga Nagar (250001), Meerut, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"965","container-title":["Current Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.eurekaselect.com\/article\/download?doi=10.2174\/0115665232413980251103133741","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.eurekaselect.com\/251454\/article","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.eurekaselect.com\/article\/download?doi=10.2174\/0115665232413980251103133741","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T18:32:41Z","timestamp":1774809161000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.eurekaselect.com\/251454\/article"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3,25]]},"references-count":0,"alternative-id":["LiveAll1"],"URL":"https:\/\/doi.org\/10.2174\/0115665232413980251103133741","relation":{},"ISSN":["1566-5232"],"issn-type":[{"value":"1566-5232","type":"print"}],"subject":[],"published":{"date-parts":[[2026,3,25]]},"assertion":[{"value":"Peer 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