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Sirolimus treatment of severe PTEN hamartoma tumor syndrome: case report and in vitro studies.

Abstract:

BACKGROUND: Phosphatase and tensin homolog (PTEN) hamartoma tumor syndrome (PHTS) is caused by germ line mutations in the PTEN gene. Symptoms include cancer predisposition, immune deviations, and lipomas/lipomatosis. No causal standard therapy is available. We describe a therapeutic attempt with the mammalian target of rapamycin (mTOR) inhibitor sirolimus for a PHTS patient suffering from thymus hyperplasia and lipomatosis. We furthermore assessed the in vitro effects of sirolimus and other i...

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Publisher copy:
10.1038/pr.2013.246

Authors


Schmid, GL More by this author
Kässner, F More by this author
Körner, A More by this author
Kratzsch, J More by this author
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Publisher:
Lippincott Williams and Wilkins
Journal:
Pediatric research
Volume:
75
Issue:
4
Pages:
527-534
Publication date:
2014-04-05
DOI:
EISSN:
1530-0447
ISSN:
0031-3998
URN:
uuid:d06ef43d-3c3d-48fe-bbb3-cd172d4ebd18
Source identifiers:
459785
Local pid:
pubs:459785

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