Fig. 1: SPVN06 structure. | Gene Therapy

Fig. 1: SPVN06 structure.

From: Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies

Fig. 1

SPVN06 encodes the two synergistic isoforms of NXNL1, RdCVF and RdCVFL, in a single vector. The total size of the expression cassette is 4926 base pairs. AAV adeno-associated virus, ITR inverted terminal repeats, RdCVF rod-derived cone viability factor, RdCVFL rod-derived cone viability factor long-form, pA polyadenylation regulatory sequences.

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