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. 2023 Oct 8;11(10):2725. doi: 10.3390/biomedicines11102725

Figure 4.

Figure 4

Domain organization of subset of all-in-one rAAV vectors employed for pre-clinical in vivo gene editing therapies. (A) Functional knockout of Vegfa or Hif1a genes underlying macular degeneration based on an all-in-one AAV coding for the Campylobacter jejuni Cas9. (B) Functional knockout of a dysfunctional dystrophin gene causing Duchenne muscular dystrophy with an all-in-one rAAV vector coding for an optimized Staphylococcus aureus Cas9. (C) CRISPRa-based gene therapy of LAMA2-related muscular dystrophy with an all-in-one AAV coding for a deactivated Staphylococcus aureus Cas9 fused to the transcription factor VP64 that promotes upregulation of the Lama1 gene. (D) Correction of tyrosinemia with an all-in-one AAV that codes for a deactivated Neisseria meningitidis Cas9 fused to the adenine base editor ABE8e causing it to correct a mutation responsible for this condition. The figure was created with Biorender.