{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T03:06:44Z","timestamp":1784776004640,"version":"3.55.0"},"reference-count":51,"publisher":"American Association for Cancer Research (AACR)","issue":"7","funder":[{"DOI":"10.13039\/501100011033","name":"Agencia Estatal de Investigaci\u00f3n","doi-asserted-by":"publisher","award":["PI20\/00102"],"award-info":[{"award-number":["PI20\/00102"]}],"id":[{"id":"10.13039\/501100011033","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004837","name":"Ministerio de Ciencia e Innovaci\u00f3n","doi-asserted-by":"publisher","award":["PID2020-116184RB-I00 \/AEI\/10.13038\/501100011033"],"award-info":[{"award-number":["PID2020-116184RB-I00 \/AEI\/10.13038\/501100011033"]}],"id":[{"id":"10.13039\/501100004837","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004837","name":"Ministerio de Ciencia e Innovaci\u00f3n","doi-asserted-by":"publisher","award":["RYC-2022-035574-I"],"award-info":[{"award-number":["RYC-2022-035574-I"]}],"id":[{"id":"10.13039\/501100004837","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100015940","name":"PTEN Research","doi-asserted-by":"publisher","award":["IJC-21-001"],"award-info":[{"award-number":["IJC-21-001"]}],"id":[{"id":"10.13039\/100015940","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100020618","name":"Institut de Recerca contra la Leuc\u00e8mia Josep Carreras","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100020618","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100019179","name":"Fundaci\u00f3n Bancaria Caixa d'Estalvis i Pensions de Barcelona","doi-asserted-by":"publisher","award":["LCF\/BQ\/PR20\/11770002"],"award-info":[{"award-number":["LCF\/BQ\/PR20\/11770002"]}],"id":[{"id":"10.13039\/501100019179","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["aacrjournals.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,7,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title\/>\n                    <jats:p>PTEN hamartoma tumor syndrome (PHTS) is a rare tumor risk disorder caused by germline loss-of-function mutations in PTEN. Half of these patients develop vascular malformations, a hamartoma characterized by overgrowth of vessels. In this study, we harness biopsies and patient-derived endothelial cells (EC) to study the genetic etiology of PHTS-related vascular malformations. We discover that these lesions are generated by somatic loss of the PTEN wild-type allele through copy-neutral loss of heterozygosity, leading to somatic uniparental disomy of the PTEN-mutated allele in ECs. We established a mouse model of PHTS-related vascular malformations and identified that the mTOR inhibitor rapamycin and AKT inhibitor capivasertib block vascular lesion growth. As proof-of-concept for clinical activity, off-label treatment with rapamycin of two patients with PHTS reduced vascular overgrowth and abrogated lesion-associated pain. Overall, our results uncover the genetic cause of vascular malformations in patients with PHTS and open new avenues for therapeutic intervention.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Significance:<\/jats:title>\n                    <jats:p>Somatic loss of PTEN in ECs causes vascular malformations in patients with the tumor risk syndrome PHTS. These lesions respond to PI3K signaling inhibition.<\/jats:p>\n                    <jats:p>See related commentary by Del Prior and Toker, p. 1306<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1158\/2159-8290.cd-24-0807","type":"journal-article","created":{"date-parts":[[2025,3,28]],"date-time":"2025-03-28T15:14:11Z","timestamp":1743174851000},"page":"1350-1362","update-policy":"https:\/\/doi.org\/10.1158\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Somatic Uniparental Disomy of\n                    <i>PTEN<\/i>\n                    in Endothelial Cells Causes Vascular Malformations in Patients with PTEN Hamartoma Tumor Syndrome"],"prefix":"10.1158","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7007-3155","authenticated-orcid":false,"given":"Sandra D.","family":"Castillo","sequence":"first","affiliation":[{"name":"1Endothelial Pathobiology and Microenvironment, Josep Carreras Leukaemia Research Institute, Barcelona, Spain."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-0570-1918","authenticated-orcid":false,"given":"Xabier","family":"Perosanz","sequence":"additional","affiliation":[{"name":"1Endothelial Pathobiology and Microenvironment, Josep Carreras Leukaemia Research Institute, Barcelona, Spain."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5481-2774","authenticated-orcid":false,"given":"Andrew K.","family":"Ressler","sequence":"additional","affiliation":[{"name":"2Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, North Carolina."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2500-3200","authenticated-orcid":false,"given":"Marta","family":"Ivars","sequence":"additional","affiliation":[{"name":"3Department of Dermatology, Hospital Sant Joan de D\u00e9u, Barcelona, Spain."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6844-974X","authenticated-orcid":false,"given":"Jairo","family":"Rodr\u00edguez","sequence":"additional","affiliation":[{"name":"4Research and Development Department, qGenomics Laboratory, Barcelona, Spain."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0174-674X","authenticated-orcid":false,"given":"Carlota","family":"Rovira","sequence":"additional","affiliation":[{"name":"5Department of Pathology, Hospital Sant Joan de D\u00e9u Barcelona, Barcelona, Spain."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2713-3383","authenticated-orcid":false,"given":"Emanuele M.","family":"Nola","sequence":"additional","affiliation":[{"name":"1Endothelial Pathobiology and Microenvironment, Josep Carreras Leukaemia Research Institute, Barcelona, 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