Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2

Deficiency of adenosine deaminase 2 (DADA2) is a monogenic autoinflammatory disorder characterized by systemic inflammation, vasculopathy, immunodeficiency, and bone marrow failure. Current therapies—including anti-TNF agents and allogeneic hematopoietic stem cell transplantation (HSCT)—have limitat...

وصف كامل

التفاصيل البيبلوغرافية

المؤلفون الرئيسيون:	Chiara Rigamonti, Dimitri Bulté, Federica Barzaghi, Cristina Mesa-Nuñez, Luca Basso-Ricci, Emanuela Pettinato, Camilla Visconti, Sara Degl’Innocenti, Francesco Gazzo, Fabrizio Benedicenti, Sergio Arévalo, Pamela Quaranta, Francesca Sanvito, Roberta Caorsi, Francesca Schena, Michela Lupia, Silvia Federici, Antonella Insalaco, Fabrizio De Benedetti, Serena Scala, Maurizio Miano, Francesca Conti, Maria Pia Cicalese, Eugenio Montini, Marco Gattorno, Carlo Dufour, Alessandro Aiuti, Alessandra Mortellaro
التنسيق:	Artigo
اللغة:	Inglês
منشور في:	Elsevier 2025-12-01
سلاسل:	Molecular Therapy: Methods & Clinical Development
الموضوعات:	deficiency of adenosine deaminase 2 gene therapy lentiviral vector hematopoietic stem cells preclinical development
الوصول للمادة أونلاين:	http://www.sciencedirect.com/science/article/pii/S2329050125002190
الوسوم:	إضافة وسم لا توجد وسوم, كن أول من يضع وسما على هذه التسجيلة!