Fig. 1
From: A one-base therapeutic insertion in the HBG2 distal promoter reactivates γ-globin expression
Introducing insA elevates γ-globin expression in HUDEP-2. (a) Indel distribution of HBG2 edited in HUDEP-2 cells analyzed by Synthego webtool after Sanger sequencing. (b-c) Quantitative real-time PCR and western blot analysis of insA HUDEP-2 cells before differentiation. (d-f) Quantitative real-time PCR (d-e) and western blot (f) analysis of insA and insA KO HUDEP-2 clones after 8 days differentiation. (g) Flow cytometry was performed with anti-HbF antibody to detect the HbF cells in insA and insA KO clones. Ctrl indicated control cells edited in the AAVS1 locus. Data from ≥3 independent experiments are presented as mean ± SD (*p < 0.05, ****p < 0.0001)