A5018306893- Hematopoietic Stem Cell Transplantation
- RNA Interference and Gene Delivery
- Virus-based gene therapy research
- CRISPR and Genetic Engineering
- T-cell and B-cell Immunology
- Single-cell and spatial transcriptomics
- Immune Cell Function and Interaction
- Cancer Genomics and Diagnostics
- Viral Infectious Diseases and Gene Expression in Insects
Fred Hutch Cancer Center
2021-2023
Clinical applications of hematopoietic stem cell (HSC) gene editing are limited due to their complex and expensive logistics. HSC is commonly performed ex vivo using electroporation requires good manufacturing practice (GMP) facilities, similar bone marrow transplant centers. In could overcome this limitation; however, unsuitable for systemic in HSCs. Here we evaluated polymer-based nanoparticles (NPs), which also be used administration, the delivery mRNA nucleases human granulocyte...
Hematopoietic stem cell (HSC) gene therapy is currently performed on CD34
ABSTRACT Reconstitution after hematopoietic stem cell (HSC) transplantation is assumed to occur in two distinct phases: initial recovery mediated by short-term progenitors and long-term repopulation multipotent HSCs which do not contribute reconstitution during the first 6-9 months. We have previously reported exclusive engraftment of HSC-enriched CD34 + CD45RA - CD90 phenotype a nonhuman primate model. Here, we closely followed clonal diversity kinetics these animals. Enhanced sampling high...