{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T23:17:12Z","timestamp":1789514232003,"version":"build-2803163510"},"reference-count":56,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2020,10,31]],"date-time":"2020-10-31T00:00:00Z","timestamp":1604102400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100005010","name":"Associazione Italiana per la Ricerca sul Cancro","doi-asserted-by":"publisher","award":["23329"],"award-info":[{"award-number":["23329"]}],"id":[{"id":"10.13039\/501100005010","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Associazione Nazionale Atassia telangiectasia","award":["ANAt 2019"],"award-info":[{"award-number":["ANAt 2019"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cancers"],"abstract":"<jats:p>Ataxia-Telangiectasia (A-T) is a rare autosomal recessive disorder, first reported in 1926, caused by a deficiency of ATM (Ataxia-Telangiectasia Mutated) protein. The disease is characterized by progressive cerebellar neurodegeneration, immunodeficiency, leukemia, and lymphoma cancer predisposition. Immunoglobulin replacement, antioxidants, neuroprotective factors, growth, and anti-inflammatory hormones are commonly used for A-T treatment, but, to date, there is no known cure. Bone marrow transplantation (BMT) is a successful therapy for several forms of diseases and it is a valid approach for tumors, hemoglobinopathies, autoimmune diseases, inherited disorders of metabolism, and other pathologies. Some case reports of A-T patients have shown that BMT is becoming a good option, as a correct engraftment of healthy cells can restore some aspects of immunologic capacity. However, due to a high risk of mortality as a result of a clinical and cellular hypersensitivity to ionizing radiation and radiomimetic drugs, a specific non-myeloablative conditioning is required before BMT. Although BMT might be considered as one promising therapy for the treatment of immunological defects and cancer prevention in selected A-T patients, the therapy is currently not recommended or recognized and the eligibility of A-T patients for BMT is a point to deepen and deliberate.<\/jats:p>","DOI":"10.3390\/cancers12113207","type":"journal-article","created":{"date-parts":[[2020,10,31]],"date-time":"2020-10-31T21:39:56Z","timestamp":1604180396000},"page":"3207","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review"],"prefix":"10.3390","volume":"12","author":[{"given":"Bruna","family":"Sabino Pinho de Oliveira","sequence":"first","affiliation":[{"name":"Department of Anatomical, Histological, Forensic Medicine and Orthopedic Sciences, Faculty of Pharmacy and Medicine, Sapienza University, Via Scarpa 16, 00161 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabrina","family":"Putti","sequence":"additional","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, IBBC-CNR, Campus Adriano Buzzati Traverso, Via Ramarini 32, 00015 Monterotondo, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0572-5900","authenticated-orcid":false,"given":"Fabio","family":"Naro","sequence":"additional","affiliation":[{"name":"Department of Anatomical, Histological, Forensic Medicine and Orthopedic Sciences, Faculty of Pharmacy and Medicine, Sapienza University, Via Scarpa 16, 00161 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6387-5187","authenticated-orcid":false,"given":"Manuela","family":"Pellegrini","sequence":"additional","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, IBBC-CNR, Campus Adriano Buzzati Traverso, Via Ramarini 32, 00015 Monterotondo, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2020,10,31]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"572","DOI":"10.1182\/blood-2003-12-4226","article-title":"Bone marrow transplantation restores immune system function and prevents lymphoma in Atm-deficient mice","volume":"104","author":"Bagley","year":"2004","journal-title":"Blood"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1187","DOI":"10.1016\/j.dnarep.2004.04.010","article-title":"Ataxia-telangiectasia, an evolving phenotype","volume":"3","author":"Chun","year":"2004","journal-title":"DNA Repair"},{"key":"ref_3","first-page":"112","article-title":"Localization of an ataxia-telangiectasia gene to an \u2212500 kb interval on chromosome 11q23.1: Linkage analysis of 176 families by an international consortium","volume":"57","author":"Lange","year":"1995","journal-title":"Am. 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