Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis

Articolo
Data di Pubblicazione:
2018
Citazione:
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis / Tucci, Francesca; Calbi, Valeria; Barzaghi, Federica; Migliavacca, Maddalena; Ferrua, Francesca; Bernardo, Maria Ester; Canarutto, Daniele; Consiglieri, Giulia; Recupero, Salvatore; Calzatini, Francesco; Gabaldo, Michela; Lucano, Caterina; Casiraghi, Miriam; Darin, Silvia; Dionisio, Francesca; Marktel, Sarah; Cestone, Enza; Finazzi, Renato; Mieli-Vergani, Giorgina; Boeri, Enzo; Appleby, Jonathan; Elaziz, Dalia Abd; Ciceri, Fabio; Aiuti, Alessandro; Cicalese, Maria Pia. - In: HEPATOLOGY. - ISSN 0270-9139. - 68:6(2018), pp. 2434-2437. [10.1002/hep.30160]
Abstract:
Patients with inborn error diseases can be candidates for autologous haematopoietic stem cells (HSC) gene therapies (GT) but may require negative viral screening, including Hepatitis C (HCV), to allow HSC manipulation in Good Manufacturing Practices areas. In case of HCV positivity, patients might be excluded from life-saving treatments. As HCV antibodies could be negative in young infant immunodeficient patients due to their immature/impaired immune system, or positive due to maternal-fetal antibody transmission, the risk is usually also evaluated on the basis of the HCV-RNA. This article is protected by copyright. All rights reserved.
Tipologia CRIS:
1.1 Articolo in rivista
Keywords:
Adenosine Deaminase; Agammaglobulinemia; Antiviral Agents; Benzimidazoles; Fluorenes; Hepatitis C; Humans; Infant; Male; Severe Combined Immunodeficiency; Uridine Monophosphate; Genetic Therapy
Elenco autori:
Autori di Ateneo:
Link alla scheda completa:
https://iris.unisr.it/handle/20.500.11768/106182
Pubblicato in:
HEPATOLOGY
Journal