Fig. 1: Programming RfxCas13d to target ataxin-2.
From: Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins
a Overview of the strategy for targeting ATXN2 using RfxCas13d. b Schematic of the mature mouse ataxin-2 (mATXN2) mRNA and location of the crRNA (CRISPR RNA) binding sites (blue bars). c EGFP mean fluorescence intensity (MFI) in HEK293T cells transfected with mATXN2-T2A-EGFP alongside RfxCas13d and each mATXN2-targeting crRNA. Data are normalized to MFI from cells transfected with mATXN2-T2A-EGFP, RfxCas13d, and a non-targeted (NTG) crRNA (n = 3). d, e Relative (d) mATXN2 mRNA and (e) ataxin-2 protein from Neuro2A cells transfected with RfxCas13d and crRNA-10 at 48 hr post-transfection. Data are normalized to cells transfected with RfxCas13d and a NTG crRNA (n = 4 for mRNA, n = 3 for protein). All data points are biologically independent samples. Values represent means and error bars indicate SD. Data were compared using a one-tailed unpaired t-test, with the exact P-values shown.
