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J Neurol Sci ; 335(1-2): 134-8, 2013 Dec 15.
Artigo em Inglês | MEDLINE | ID: mdl-24090759

RESUMO

Ataxia telangiectasia (AT) and ataxia oculomotor apraxia type 2 (AOA2) are autosomal recessive ataxias caused by mutations in genes involved in maintaining DNA integrity. Lifespan in AT is greatly shortened (20s-30s) due to increased susceptibility to malignancies (leukemia/lymphoma). Lifespan in AOA2 is uncertain. We describe a woman with variant AT with two novel mutations in ATM (IVS14+2T>G and 5825C>T, p.A1942V) who died at age 48 with pancreatic adenocarcinoma. Her mutations are associated with an unusually long life for AT and with a cancer rarely associated with that disease. We also describe two siblings with AOA2, heterozygous for two novel mutations in senataxin (3 bp deletion c.343-345 and 1398T>G, p.I466M) who have survived into their 70s, allowing us to characterize the longitudinal course of AOA2. In contrast to AT, we show that persons with AOA2 can experience a prolonged lifespan with considerable motor disability.


Assuntos
Ataxia Telangiectasia/genética , Mutação/genética , RNA Helicases/genética , Adulto , Idoso , Ataxia Telangiectasia/mortalidade , Ataxia Telangiectasia/patologia , Encéfalo/metabolismo , Encéfalo/patologia , Calbindinas/metabolismo , DNA Helicases , Feminino , Estudos de Associação Genética , Humanos , Masculino , Enzimas Multifuncionais , Análise de Sobrevida
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