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

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,...

Full description

Saved in:
Bibliographic Details
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
Series:Molecular Therapy: Nucleic Acids
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
Tags: Add Tag
No Tags, Be the first to tag this record!

Internet

http://www.sciencedirect.com/science/article/pii/S216225312400310X

Similar Items