BCL11A enhancer–edited hematopoietic stem cells persist in rhesus monkeys without toxicity

Selami Demirci, Jing Zeng, Yuxuan Wu, +24

Journal of Clinical Investigation

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

Naoya Uchida, Claire Drysdale, Tina Nassehi, +9

Molecular Therapy — Methods & Clinical Development

High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2

Naoya Uchida, Tina Nassehi, Claire Drysdale, +7

Molecular Therapy — Methods & Clinical Development

Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model

Naoya Uchida, Tina Nassehi, Claire Drysdale, +10

Molecular Therapy

Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice

Alexis Leonard, Morgan Yapundich, Tina Nassehi, +7

Molecular Therapy — Methods & Clinical Development

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Naoya Uchida, Linhong Li, Tina Nassehi, +15

Cell Reports Medicine

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

Selami Demirci, Bjorg Gudmundsdottir, Quan Li, +8

Molecular Therapy — Methods & Clinical Development

Sustained fetal hemoglobin induction in vivo is achieved by <i>BCL11A</i> interference and coexpressed truncated erythropoietin receptor

Naoya Uchida, Francesca Ferrara, Claire Drysdale, +17

Science Translational Medicine