Fig. 6: In vivo applications of R26SAM gene modulation. | Nature Communications

Fig. 6: In vivo applications of R26SAM gene modulation.

From: Tissue-specific activation of gene expression by the Synergistic Activation Mediator (SAM) CRISPRa system in mice

Fig. 6: In vivo applications of R26SAM gene modulation.

a Schematic of murine Ttr replaced with WT human TTR sequence. Mouse sequences are represented by blue lines and human sequences by purple. b Circulating mouse and human TTR levels were determined by species-specific ELISA and plotted as mean +/− SD (n = 8). Commercial normal human serum (NHS) was run as a control for normal human expression levels (n = 1). Humanized allele vs NHS: p = 0.0071. c Homozygous humanized TTR mice were crossed to R26SAM/SAM mice and injected with AAV83aTtr or AAV8GFP (n = 4). Asterisks (*) indicates significance, and the number of asterisks indicates the number of 0s after the decimal point. One-tailed, unpaired Student’s t-test for (b, c).

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