Côd QR

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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Elsevier 2025-12-01
Cyfres:Molecular Therapy: Methods & Clinical Development
Pynciau:
deficiency of adenosine deaminase 2
gene therapy
lentiviral vector
hematopoietic stem cells
preclinical development
Mynediad Ar-lein:http://www.sciencedirect.com/science/article/pii/S2329050125002190
Tagiau: Ychwanegu Tag
Dim Tagiau, Byddwch y cyntaf i dagio'r cofnod hwn!

Eitemau Tebyg