{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,17]],"date-time":"2026-05-17T04:15:28Z","timestamp":1778991328023,"version":"3.51.4"},"reference-count":15,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2016,7,22]],"date-time":"2016-07-22T00:00:00Z","timestamp":1469145600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2017,1,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n               <jats:sec>\n                  <jats:title>\u2003<\/jats:title>\n                  <jats:p>In osteopetrosis, osteoclast dysfunction can lead to deafness, blindness, bone marrow failure, and death. Hematopoietic cell transplantation (HCT) is currently the only curative treatment, but outcome remains disappointing. Although a rapid progression toward HCT is detrimental to prevent further progress of disease manifestations, 70% of cases lack an HLA-matched sibling and require alternative stem cell sources. We present two cases of osteopetrosis that successfully received an HCT with haploidentical TcR\u03b1\u03b2-depleted cells from one of the parents. These cases showed no further disease progression, had restoration of functional osteoclasts, and illustrate this approach to enable prompt HCT with ready available parental donors and rapid and sustained hematological, including osteoclast, recovery. \u00a9 2016 American Society for Bone and Mineral Research.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1002\/jbmr.2921","type":"journal-article","created":{"date-parts":[[2016,7,22]],"date-time":"2016-07-22T20:06:47Z","timestamp":1469218007000},"page":"82-85","source":"Crossref","is-referenced-by-count":8,"title":["Transplantation of Haploidentical TcRa\u00df-Depleted Hematopoietic Cells Allows for Optimal Timing and Sustained Correction of the Metabolic Defect in Children With Infantile Osteopetrosis"],"prefix":"10.1093","volume":"32","author":[{"given":"Cornelis JH","family":"Pronk","sequence":"first","affiliation":[{"name":"Lund University, Institution for Laboratory MedicineDivision of Molecular HematologyLundSweden"},{"name":"Department of Pediatric Oncology\/HematologySk\u00e5ne University HospitalLundSweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dominik","family":"Turkiewicz","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/HematologySk\u00e5ne University HospitalLundSweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristina","family":"Vult von Steyern","sequence":"additional","affiliation":[{"name":"Center for Medical Imaging and PhysiologySk\u00e5ne University HospitalLundSweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mats","family":"Ehinger","sequence":"additional","affiliation":[{"name":"Department of PathologySk\u00e5ne University HospitalLundSweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Josefina","family":"Dykes","sequence":"additional","affiliation":[{"name":"Department of Clinical Immunology and Transfusion MedicineOffice of Medical ServicesLundSweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jacek","family":"Toporski","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/HematologySk\u00e5ne University HospitalLundSweden"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2016,7,22]]},"reference":[{"issue":"9","key":"2024021014255829400_jbmr2921-bib-0001","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1038\/nrendo.2013.137","article-title":"Osteopetrosis: genetics, treatment and new insights into osteoclast function","volume":"9","author":"Sobacchi","year":"2013","journal-title":"Nat Rev Endocrinol"},{"issue":"13","key":"2024021014255829400_jbmr2921-bib-0002","doi-asserted-by":"crossref","first-page":"701","DOI":"10.1056\/NEJM198003273021301","article-title":"Successful bone-marrow transplantation for infantile malignant 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