Restoring hematopoietic stem and progenitor cell function in Fancc−/− mice by in situ delivery of RNA lipid nanoparticles

Fanconi anemia (FA) is a congenital multisystem disorder characterized by early-onset bone marrow failure (BMF) and cancer susceptibility. While ex vivo gene addition and repair therapies are being considered as treatment options, depleted hematopoietic stem cell (HSC) pools, poor HSC mobilization,...

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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Omar Banda, Sarah E. Adams, Linah Omer, Seul K. Jung, Hooda Said, Theerapat Phoka, Ying Tam, Drew Weissman, Stefano Rivella, Mohamad-Gabriel Alameh, Peter Kurre
Format:	Article
Language:	English
Published:	Elsevier 2025-03-01
Subjects:	MT: Delivery Strategies lipid nanoparticles Fanconi anemia monogenic disorders protein replacement therapy in vivo delivery
Online Access:	http://www.sciencedirect.com/science/article/pii/S216225312400310X