Biblio
RhoH Participates in a Multi-Protein Complex with the Zinc Finger Protein Kaiso That Regulates Both Cytoskeletal Structures and Chemokine-Induced T Cells. Small GTPases. 2016.
. Restored macrophage function ameliorates disease pathophysiology in a mouse model for IL10 receptor deficient very early onset inflammatory bowel disease. J Crohns Colitis. 2021.
. Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.
Modulating the stem cell niche for tissue regeneration. Nat Biotechnol. 2014.
. The long road traveled in hematopoietic stem cell gene therapy. Mol Ther. 2022.
. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016.
Investigational curative gene therapy approaches to sickle cell disease. Blood Adv. 2021;5(23):5452.
. Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy. Nat Commun. 2023;14(1):1900.
Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): An initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Pediatr Blood Cancer. 2013.
. Clonal hematopoiesis in sickle cell disease. J Clin Invest. 2022.
The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020.
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