{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,30]],"date-time":"2026-06-30T22:10:20Z","timestamp":1782857420229,"version":"3.54.5"},"reference-count":45,"publisher":"MDPI AG","issue":"4","license":[{"start":{"date-parts":[[2025,4,18]],"date-time":"2025-04-18T00:00:00Z","timestamp":1744934400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Regenerative Medicine Acceleration Foundation under the Ministry of Health and Welfare, Republic of Korea","award":["R\u20133-0005"],"award-info":[{"award-number":["R\u20133-0005"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Children"],"abstract":"<jats:p>Background\/Objectives: Hutchinson\u2013Gilford progeria syndrome (HGPS) is a rare genetic disorder that cause premature aging due to LMNA mutations and progerin accumulation. Although lonafarnib, an FDA-approved farnesyltransferase inhibitor, offers modest extension of life, the disease remains progressive. As progeria is associated with stem cell depletion and mesenchymal stem cell (MSC) therapy has shown efficacy in treating atherosclerosis, we aimed to evaluate its efficacy and safety in HGPS. Methods: A 7-year-old male with classic HGPS and preexisting severe cerebrovascular disease received four intravenous infusion of bone marrow-derived MSCs (2.5 \u00d7 10\u2075 cells\/kg) over 8 months. Growth, metabolic, cardiovascular, musculoskeletal, auditory, and inflammatory cytokines were monitored throughout the study. Prophylactic enoxaparin was administered to prevent vascular complications. Results: MSC therapy was associated with improved lean body mass (11.5%), bone mineral density (L-spine z-score: 0.55 \u2192 2.03), reduced arterial stiffness (9.98% reductionin pulse wave velocity), joint range of motion, dentition, and decreased sICAM-1 levels. However, Cardiovascular deterioration continued, and the patient passed away 10 months after the fourth dose, likely due to progression of the underlying vascular disease. No severe adverse effects were attributed to MSC therapy. Conclusions: MSC therapy may offer short-term benefits in arterial stiffness, bone health and inflammation in HGPS without notable safety concerns. Further studies are warranted to validate these findings, explore earlier intervention, and determine long-term efficacy and optimal dosing strategies.<\/jats:p>","DOI":"10.3390\/children12040523","type":"journal-article","created":{"date-parts":[[2025,4,17]],"date-time":"2025-04-17T23:16:46Z","timestamp":1744931806000},"page":"523","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Mesenchymal Stem Cell Therapy for Hutchinson\u2013Gilford Progeria: Improvements in Arterial Stiffness and Bone Mineral Density in a Single Case"],"prefix":"10.3390","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0009-0002-5972-6191","authenticated-orcid":false,"given":"Eun-Young","family":"Joo","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Inha University College of Medicine, Incheon 22212, Republic of Korea"},{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Advanced Regenerative Medicine Clinical Trial Center, Inha University Hospital, Incheon 22332, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3793-6577","authenticated-orcid":false,"given":"Ji-Sun","family":"Park","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Inha University College of Medicine, Incheon 22212, Republic of Korea"},{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Advanced Regenerative Medicine Clinical Trial Center, Inha University Hospital, Incheon 22332, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1799-5860","authenticated-orcid":false,"given":"Hyun-Tae","family":"Shin","sequence":"additional","affiliation":[{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Advanced Regenerative Medicine Clinical Trial Center, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Department of Dermatology, Inha University College of Medicine, Incheon 22212, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Myungji","family":"Yoo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Inha University College of Medicine, Incheon 22212, Republic of Korea"},{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Advanced Regenerative Medicine Clinical Trial Center, Inha University Hospital, Incheon 22332, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0893-0512","authenticated-orcid":false,"given":"Su-Jin","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Inha University College of Medicine, Incheon 22212, Republic of Korea"},{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Advanced Regenerative Medicine Clinical Trial Center, Inha University Hospital, Incheon 22332, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7386-0015","authenticated-orcid":false,"given":"Ji-Eun","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Inha University College of Medicine, Incheon 22212, Republic of Korea"},{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Advanced Regenerative Medicine Clinical Trial Center, Inha University Hospital, Incheon 22332, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5766-0179","authenticated-orcid":false,"given":"Gwang-Seong","family":"Choi","sequence":"additional","affiliation":[{"name":"Gyeonggi-Incheon Regional Specialized Rare Disease Institute, Inha University Hospital, Incheon 22332, Republic of Korea"},{"name":"Department of Dermatology, Inha University College of Medicine, Incheon 22212, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,4,18]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2055","DOI":"10.1126\/science.1084125","article-title":"Lamin a truncation in Hutchinson\u2013Gilford 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