Fig. 1: PE induced efficient and precise gene editing in rabbit.
From: Efficient and precise generation of Tay–Sachs disease model in rabbit by prime editing system
a The base insertion efficiency of PE system in HEK293FT cells. b PE induced efficient and precise gene editing in rabbit embryos. c Editing frequency (HEXA ins TATC) of PegRNA screening with PBS length (8–16 nt) in rabbit embryos. d Editing frequency (HEXA ins TATC) of PegRNA screening with RT template length (10–18 nt) in rabbit embryos. e Editing frequency (HEXA ins TATC) of CRISPR–Cas9 system-mediated HDR compare with PE3. f The target sequence at HEXA locus by PE system. The PAM and sgRNA target sequences are shown in green and black, target mutation (red), frameshift mutation leads to PTC mutation (red and red star). g Editing frequency determination of HEXA ins TATC rabbit by deep sequencing. h Expression of HEXA gene was determined by qRT-PCR. i HEXA protein was determined by western blot. j X-ray radiography of WT and HEXA ins TATC rabbits. Red circle, increased cervical lordosis; Red arrows, clasping of the limbs. k Masson’s trichrome staining of gastrocnemius from WT and HEXA ins TATC rabbits. Blue arrow highlights the myopathy with fibrosis and inflammatory cell infiltration. l HE staining of hippocampus from WT and HEXA heterozygous rabbits. The red arrow highlights the enlargement of perineural space.
