Figure 1 | Scientific Reports

Figure 1

From: Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators

Figure 1

Experimental workflow. (A) Mice were intravitreally injected with an AAV2-vector in both eyes at P21, and the retinal ganglion cell (RGC) number was quantified 3 months later. An experimental or control virus was injected into the vitreous humor of the mouse eye, where it can diffuse into the retina and target RGCs. (B) FD mutant mice were divided randomly into two groups: control and experimental. The control group was fed a standard chow diet, and the experimental group with a specially formulated chow diet containing a novel splicing modulator compound, PTC680. Groups were started on treatment at three different ages: E18.5, P21 or P90. After 3 months on either the control or experimental diet, retinas were analyzed and quantified for exon 20 inclusion and RGC numbers, respectively. Created with BioRender.com.

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