A5037012882- T-cell and B-cell Immunology
- Immune Cell Function and Interaction
- CRISPR and Genetic Engineering
- Transgenic Plants and Applications
- Cytomegalovirus and herpesvirus research
- CAR-T cell therapy research
- Virus-based gene therapy research
- Viral Infectious Diseases and Gene Expression in Insects
Stanford University
2020-2024
Gene editing of FOXP3 ensures regulated expression and restored function in T cells, supporting clinical applicability.
Abstract Genome editing by homology directed repair (HDR) is leveraged to precisely modify the genome of therapeutically relevant hematopoietic stem and progenitor cells (HSPCs). Here, we present a new approach increasing frequency HDR in human HSPCs delivery an inhibitor 53BP1 (named “i53”) as recombinant peptide. We show that use i53 peptide effectively increases HDR-mediated at variety loci well other primary cell types. incorporating protein allows high frequencies while lowering amounts...
Forkhead box P3 (FOXP3) is an essential transcription factor for regulatory T cell (Treg) function. Defects in Tregs mediate many immune diseases including the monogenic autoimmune disease dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX), which caused by FOXP3 mutations. Treg products are a promising modality to induce allograft tolerance or reduce use of immunosuppressive drugs prevent rejection, as well treatment acquired diseases. We have recently opened phase I...
Abstract Genome editing by homology directed repair (HDR) is leveraged to precisely modify the genome of therapeutically relevant hematopoietic stem and progenitor cells (HSPCs). Here, we present a new approach increasing frequency HDR in human HSPCs delivery i53 recombinant protein. We show that use peptide effectively increases HDR-mediated at variety loci as well other primary cell types. incorporating protein allows high levels be attained while lowering amounts AAV6 needed 8-fold....