WebApr 10, 2024 · These cells have been used in gene editing and hematopoietic differentiation studies [8,16,17,18,19,20]. Considering that most preclinical studies on SCD drug discovery have used immortalized CD34 + cells or animal models, the standardization of efficient hematopoietic differentiation protocols from patient-derived iPSCs would be … WebDec 9, 2024 · Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs Mol Ther Nucleic Acids. 2024 Dec 9;31:105-121. doi: 10.1016/j.omtn.2024.12.006. eCollection 2024 Mar 14. Authors
Therapeutic base editing of human hematopoietic stem cells
WebAssistant Manager (IPR - Biosimilars/Biotech) at Intas Pharmaceuticals Ltd & Registered Indian Patent Agent (IN/PA-3349) 1w WebWe are developing improved Cas9 proteins for delivery ex vivo into CD34+ HSPCs for therapeutic application to sickle cell disease and beta-thalassemia. The goal is to modify the hematopoietic stem cells of a patient to complement the loss of function of beta-globin gene and then return these cells to the patient through an autologous transplant. diamonds coffee shop minneapolis
Optimizing CD34+ Cultures and Gene Editing For Efficiency
WebCommon methods of genome editing utilize the homology-directed repair (HDR) pathway. However, these approaches are inefficient in human hematopoietic stem and progenitor cells (HSPCs), where non … WebDec 5, 2024 · We assessed the frequency of gene editing as-sociated with CTX001 in CD34+ HSPCs obtained from 10 healthy donors. High frequencies of al-lelic editing (mean [±SD], 80±6%) were observed and ... WebMay 23, 2024 · Recently, we optimized conditions for high-efficiency SpCas9 RNP editing of CD34 + HSPCs by electroporation. 6 ... Therapeutic gene editing of IVS1-110G>A. (A) Schema of IVS1-110G>A mutation within HBB intron 1 and therapeutic editing strategy. (B) Indicated donors and sgRNAs used for therapeutic editing. Five days after RNP … diamonds club köln