Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin)

INTRODUCTION: β-thalassemia is caused by autosomal mutations in the β-globin gene, which induce the absence or low-level synthesis of β-globin in erythroid cells. It is widely accepted that a high production of fetal hemoglobin (HbF) is beneficial for patients with β-thalassemia. Sirolimus, also kno...

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Detalles Bibliográficos
Autores principales: Zuccato, Cristina, Cosenza, Lucia Carmela, Zurlo, Matteo, Gasparello, Jessica, Papi, Chiara, D’Aversa, Elisabetta, Breveglieri, Giulia, Lampronti, Ilaria, Finotti, Alessia, Borgatti, Monica, Scapoli, Chiara, Stievano, Alice, Fortini, Monica, Ramazzotti, Eric, Marchetti, Nicola, Prosdocimi, Marco, Gamberini, Maria Rita, Gambari, Roberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2022
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9218916/
https://www.ncbi.nlm.nih.gov/pubmed/35755297
http://dx.doi.org/10.1177/20406207221100648

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9218916/
https://www.ncbi.nlm.nih.gov/pubmed/35755297
http://dx.doi.org/10.1177/20406207221100648

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