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1. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

2. Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models

3. Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation

4. Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells

5. A macaque clonal hematopoiesis model demonstrates expansion of TET2-disrupted clones and utility for testing interventions

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

8. Intrabone transplantation of CD34+ cells with optimized delivery does not enhance engraftment in a rhesus macaque model

11. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

21. CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model

23. 291 - Optimization of Autologous Hematopoietic Progenitor Stem Cell Apheresis Collection from Plerixafor-Mobilized Patients with Sickle Cell Disease

25. A Single Dose of CD117 Antibody Drug Conjugate Enables Hematopoietic Stem Cell Based Gene Therapy in Nonhuman Primates

26. Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

34. Discordance Between Peripheral Blood and Tissue CD4+ Lymphocyte Reconstitution In Rhesus Monkeys Following Autologous Hematopoietic CD34+ Cell Transplantation: An In Vivo imaging Study

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