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.
Restored macrophage function ameliorates disease pathophysiology in a mouse model for IL10 receptor deficient very early onset inflammatory bowel disease. J Crohns Colitis. 2021..