AAV2-4D3(E)-mCherry

AAV2-4D3(E)-mCherry is an experimental gene therapy vector utilizing an adeno-associated virus serotype 2 (AAV2) capsid to deliver a genetic payload encoding a modified peptide fragment, 4D3(E), fused to an mCherry fluorescent reporter. The 4D3 peptide is derived from the phosphodiesterase 4D3 (PDE4D3) binding domain of the scaffold protein mAKAPα (muscle-selective A-kinase anchoring protein, also known as AKAP6). The 'E' variant represents a specific modification of this peptide sequence. The therapeutic mechanism involves the competitive displacement of the enzyme PDE4D3 from the mAKAPα signalosome located at the perinuclear membrane of retinal ganglion cells (RGCs). By preventing the localized degradation of cyclic AMP (cAMP) by PDE4D3, the vector increases perinuclear cAMP concentrations, which activates pro-survival and pro-regenerative signaling pathways. This approach is being investigated for its potential to provide neuroprotection and stimulate axon regeneration in conditions such as glaucoma and traumatic optic neuropathy.

Other names

AAV2-mAKAP-4D3(E)-mCherryAAV2-mCherry-4D3(E)