Эксагамглоген аутотемцел

Rec. INN (наименование, зарегистрированное ВОЗ)

DB15572

N (не классифицировано FDA)

Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of y-globin gene transcription.

Поделиться этой страницей