Утигамглоген аутогедтемцел

Prop. INN (наименование, предложенное ВОЗ)

autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) derived from apheresis peripheral blood collection of transfusion dependent β-thalassemia patients, genetically modified at the +58 erythroid specific enhancer region of the BCL11A gene by CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein 9) gene editing. The Streptococcus pyogenes Cas9 protein (spCas9) and gRNA are electroporated into the cells as a ribonucleoprotein (RNP) complex. The spCas9 sequence is appended on the N-terminus to include a Myc-like nuclear localisation signal (NLS) and appended on the C terminus to include NLS sequences derived from simian virus 40 (SV40) and nucleoplasmin.
The cell suspension was enriched in CD34+ cells using immunomagnetic microbeads, gene-edited and then cultured in the presence of media containing FMS-like tyrosine kinase 3 ligand (Flt-3L), thrombopoietin (TPO) and stem cell factor (SCF). The final substance contains ≥70% CD34+ cells

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