Use of HSC-targeted LNP to generate a mouse model of lethal alpha-thalassemia and treatment via lentiviral gene therapy. Chappell ME, Breda L, Tricoli L, Guerra A, et al. Blood 2024;144:1633-1645.

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Amedeo Smart

Independent Medical Education

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Chappell ME, Breda L, Tricoli L, Guerra A, et al. Use of HSC-targeted LNP to generate a mouse model of lethal alpha-thalassemia and treatment via lentiviral gene therapy. Blood 2024;144:1633-1645.
PMID: 38949981