Biblio
CD105 is a surface marker for receptor-targeted gene transfer into human long-term repopulating hematopoietic stem cells. Stem Cells Dev. 2014.
. Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial. Biochem Pharmacol. 2019:113711.
. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016.
Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.
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