A5080776209- CRISPR and Genetic Engineering
- Immune Cell Function and Interaction
- Cytomegalovirus and herpesvirus research
- Lysosomal Storage Disorders Research
- CAR-T cell therapy research
- Hematopoietic Stem Cell Transplantation
- Virus-based gene therapy research
- Prenatal Screening and Diagnostics
- Cellular transport and secretion
- T-cell and B-cell Immunology
- Microbial Inactivation Methods
- Mesenchymal stem cell research
- Pluripotent Stem Cells Research
- Salivary Gland Disorders and Functions
Stanford University
2020-2023
Gene editing of FOXP3 ensures regulated expression and restored function in T cells, supporting clinical applicability.
Abstract Gaucher disease is a lysosomal storage disorder caused by insufficient glucocerebrosidase activity. Its hallmark manifestations are attributed to infiltration and inflammation macrophages. Current therapies for include life−long intravenous administration of recombinant orally-available glucosylceramide synthase inhibitors. An alternative approach engineer the patient’s own hematopoietic system restore expression, thereby replacing affected cells, constituting potential one-time...
Abstract Recombination-activating genes (RAG1 and RAG2) are critical for lymphoid cell development function by initiating the variable (V), diversity (D), joining (J) (V(D)J)-recombination process to generate polyclonal lymphocytes with broad antigen specificity. The clinical manifestations of defective RAG1/2 range from immune dysregulation severe combined immunodeficiencies (SCIDs), causing life-threatening infections death early in life without hematopoietic transplantation (HCT). Despite...
Recombinant adeno-associated virus (rAAV) vectors have the unique property of being able to perform genomic targeted integration (TI) without inducing a double-strand break (DSB). In order improve our understanding mechanism behind TI mediated by AAV and its efficiency, we performed an unbiased genetic screen in human cells using promoterless AAV-homologous recombination (AAV-HR) vector system. We identified that inhibition Fanconi anemia complementation group M (FANCM) protein enhanced...
ABSTRACT Recombination-activating genes ( RAG1 and RAG2 ) are critical in lymphoid cell development function for initiating the V(D)J-recombination process to generate polyclonal lymphocytes with broad antigen-specificity. Clinical manifestations of defective RAG1/2 range from immune dysregulation severe combined immunodeficiencies (SCID), causing life-threatening infections death early life absence hematopoietic transplantation (HCT). Haploidentical HCT without myeloablative conditioning...
Umbilical cord blood is an important graft source in the treatment of many genetic, hematologic, and immunologic disorders by hematopoietic stem cell transplantation. Millions units have been collected stored for clinical use since inception banking 1989. However, biomedical research has limited access to viable samples. Here, we present a cost-effective, self-sustaining model procurement fresh umbilical components purposes within hospital-affiliated academic institutions.
IntroductionUmbilical cord blood (UCB) is a vital source of hematopoietic stem and progenitor cells (HSPCs). Since 1991, UCB has been collected stored in public banks to be primarily used as for allogeneic cell transplantation (HSCT). the seminal transplant reported 1988 by Gluckman et al., contributed advances HSCT clinical trials. However, UCB-derived remain costly difficult procure pre-clinical research. Here we present protocol multidisciplinary, self-sustaining program dedicated...