Publications by authors named "Suzette Shipp"
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 to increasing the frequency of HDR in human HSPCs by the delivery of an inhibitor of 53BP1 (named "i53") as a recombinant peptide. We show that the use of i53 peptide effectively increases the frequency of HDR-mediated genome editing at a variety of therapeutically relevant loci in HSPCs as well as other primary human cell types.
View Article and Find Full Text PDFknockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products.
Mol Ther Methods Clin Dev
December 2023
Article Synopsis
- FOXP3 is a key transcription factor for regulatory T cell (Treg) function, and defects in Tregs can lead to autoimmune diseases like IPEX due to genetic mutations.
- A phase I clinical trial has been initiated for IPEX patients using engineered Treg-like cells to improve treatment outcomes and reduce the need for immunosuppressive drugs.
- A new humanized-mouse model (hu-mouse) was developed using CRISPR to knock out FOXP3, mimicking IPEX symptoms, and showed that injection of CD4 cells could restore Treg functions and control disease symptoms, making it a valuable tool for pre-clinical studies.