A5078479788- CRISPR and Genetic Engineering
- Erythropoietin and Anemia Treatment
- Pluripotent Stem Cells Research
- PARP inhibition in cancer therapy
- Virus-based gene therapy research
- Hemoglobinopathies and Related Disorders
- Phagocytosis and Immune Regulation
- Epigenetics and DNA Methylation
- HIV Research and Treatment
- Cytomegalovirus and herpesvirus research
- Single-cell and spatial transcriptomics
- Renal and related cancers
- CAR-T cell therapy research
- Erythrocyte Function and Pathophysiology
- Animal Genetics and Reproduction
- Advanced Breast Cancer Therapies
- HIV/AIDS drug development and treatment
- Iron Metabolism and Disorders
- Insect symbiosis and bacterial influences
- Virology and Viral Diseases
- Computational Drug Discovery Methods
- Immune Cell Function and Interaction
Stanford University
2019-2025
Broad Center
2023-2025
University of California, San Francisco
2023-2025
Abstract Despite their rapidly-expanding therapeutic potential, human pluripotent stem cell (hPSC)-derived therapies continue to have serious safety risks. Transplantation of hPSC-derived populations into preclinical models has generated teratomas (tumors arising from undifferentiated hPSCs), unwanted tissues, and other types adverse events. Mitigating these risks is important increase the such therapies. Here we use genome editing engineer a general platform improve future transplantation...
Blood transfusion plays a vital role in modern medicine, but frequent shortages occur. Ex vivo manufacturing of red blood cells (RBCs) from universal donor offers potential solution, yet the high cost recombinant cytokines remains barrier. Erythropoietin (EPO) signaling is crucial for RBC development, and EPO among most expensive media components. To address this challenge, we develop highly optimized small molecule-inducible synthetic receptors (synEPORs) using design-build-test cycles...
Allogeneic transplantation of CCR5 null hematopoietic stem and progenitor cells (HSPCs) is the only known cure for HIV-1 infection. However, this treatment limited because rarity CCR5-null matched donors, morbidities associated with allogeneic transplantation, prevalence strains resistant to knockout (KO) alone. Here, we propose a one-time therapy through autologous HSPCs genetically engineered ex vivo produce both KO long-term secretion potent inhibiting antibodies from B cell progeny....
Abstract Autologous transplantation of CCR5 null hematopoietic stem and progenitor cells (HSPCs) is the only known cure for HIV-1 infection. However, this treatment limited because rarity -null matched donors, morbidities associated with allogeneic transplantation, prevalence strains resistant to knockout (KO) alone. Here, we propose a one-time therapy through autologous HSPCs genetically engineered ex vivo produce both KO long-term secretion potent inhibiting antibodies from B cell progeny....
Abstract Blood transfusion plays a vital role in modern medicine. However, availability is contingent on donated blood, and frequent shortages pose significant healthcare challenge. Ex vivo manufacturing of red blood cells (RBCs) derived from universal donor O-negative pluripotent stem emerges as solution, yet the high cost recombinant cytokines required for ex erythroid differentiation remains major barrier. Erythropoietin (EPO) signaling through EPO receptor indispensable to RBC...
<title>Abstract</title> Blood transfusion plays a vital role in modern medicine. However, availability is contingent on donated blood, and frequent shortages pose significant healthcare challenge. <italic>Ex vivo </italic>manufacturing of red blood cells (RBCs) derived from universal donor O-negative pluripotent stem emerges as solution, yet the high cost recombinant cytokines required for <italic>ex vivo</italic> erythroid differentiation remains major barrier. Erythropoietin (EPO)...
<title>Abstract</title> Blood transfusion plays a vital role in modern medicine. However, availability is contingent on donated blood, and frequent shortages pose significant healthcare challenge. Ex vivo manufacturing of red blood cells (RBCs) derived from universal donor O-negative pluripotent stem emerges as solution, yet the high cost recombinant cytokines required for ex erythroid differentiation remains major barrier. Erythropoietin (EPO) signaling through EPO receptor indispensable to...
ABSTRACT β-thalassemia pathology is not only due to loss of β-globin ( HBB ), but also erythrotoxic accumulation and aggregation the binding partner, α-globin HBA1/2 ). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace entire HBA1 gene with full-length transgene in β-thalassemia-derived hematopoietic stem progenitor cells (HSPCs), which sufficient normalize β-globin:α-globin mRNA protein ratios restore functional adult hemoglobin tetramers patient-derived red...