Reporter Expression Persists 1 Year After Adeno-Associated Virus-Mediated Gene Transfer to the Optic Nerve

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

“…Regardless of the route, it is clear that transduction of M-cells is favored and occurs efficiently at higher doses of AAV vectors. We did not observe transduction after the first synapse, which is similar to results reported in mice and dogs injected with AAV (10,11) but different from studies in dogs using AAV8 (18). Nevertheless, these data support the use of cell-specific promoters for restricting transgene expression to primary outer retinal cellular targets.…”

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

“…Regardless of the route, it is clear that transduction of M-cells is favored and occurs efficiently at higher doses of AAV vectors. We did not observe transduction after the first synapse, which is similar to results reported in mice and dogs injected with AAV (10,11) but different from studies in dogs using AAV8 (18). Nevertheless, these data support the use of cell-specific promoters for restricting transgene expression to primary outer retinal cellular targets.…”

DNA nanoparticles are safe and nontoxic in non-human primate eyes

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Adeno-Associated Virus-Mediated Expression of Vascular Endothelial Growth Factor Peptides Inhibits Retinal Neovascularization in a Mouse Model of Oxygen-Induced Retinopathy

“…The greater inhibition observed our study may result from the use of longer VEGF peptides, as previous in vitro results have shown that 12-residue peptides of VEGF exon 6 retain greater antiangiogenic activity than do 7-residue peptides (Jia et al, 2001). Alternatively, because rAAV-mediated delivery of VEGF peptides offers long-term expression (Guy et al, 1999), making repeated injections unnecessary, the concentration of VEGF peptides expressed from rAAV may be either higher, more stably present, or both to yield the observed, more dramatic anti-NV effect. The bands detected by Western blots were larger than the expected size of the Myc-tagged IgK-VEGF6(121-132) or IgK-VEGF7(22-44)/8(1) peptide.…”

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

“…Regardless of the route, it is clear that transduction of M-cells is favored and occurs efficiently at higher doses of AAV vectors. We did not observe transduction after the first synapse, which is similar to results reported in mice and dogs injected with AAV (10,11) but different from studies in dogs using AAV8 (18). Nevertheless, these data support the use of cell-specific promoters for restricting transgene expression to primary outer retinal cellular targets.…”

DNA nanoparticles are safe and nontoxic in non-human primate eyes

“……”

Adeno-Associated Virus-Mediated Expression of Vascular Endothelial Growth Factor Peptides Inhibits Retinal Neovascularization in a Mouse Model of Oxygen-Induced Retinopathy

“…The greater inhibition observed our study may result from the use of longer VEGF peptides, as previous in vitro results have shown that 12-residue peptides of VEGF exon 6 retain greater antiangiogenic activity than do 7-residue peptides (Jia et al, 2001). Alternatively, because rAAV-mediated delivery of VEGF peptides offers long-term expression (Guy et al, 1999), making repeated injections unnecessary, the concentration of VEGF peptides expressed from rAAV may be either higher, more stably present, or both to yield the observed, more dramatic anti-NV effect. The bands detected by Western blots were larger than the expected size of the Myc-tagged IgK-VEGF6(121-132) or IgK-VEGF7(22-44)/8(1) peptide.…”

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

“…Regardless of the route, it is clear that transduction of M-cells is favored and occurs efficiently at higher doses of AAV vectors. We did not observe transduction after the first synapse, which is similar to results reported in mice and dogs injected with AAV (10,11) but different from studies in dogs using AAV8 (18). Nevertheless, these data support the use of cell-specific promoters for restricting transgene expression to primary outer retinal cellular targets.…”

DNA nanoparticles are safe and nontoxic in non-human primate eyes

“……”

Adeno-Associated Virus-Mediated Expression of Vascular Endothelial Growth Factor Peptides Inhibits Retinal Neovascularization in a Mouse Model of Oxygen-Induced Retinopathy

“…The greater inhibition observed our study may result from the use of longer VEGF peptides, as previous in vitro results have shown that 12-residue peptides of VEGF exon 6 retain greater antiangiogenic activity than do 7-residue peptides (Jia et al, 2001). Alternatively, because rAAV-mediated delivery of VEGF peptides offers long-term expression (Guy et al, 1999), making repeated injections unnecessary, the concentration of VEGF peptides expressed from rAAV may be either higher, more stably present, or both to yield the observed, more dramatic anti-NV effect. The bands detected by Western blots were larger than the expected size of the Myc-tagged IgK-VEGF6(121-132) or IgK-VEGF7(22-44)/8(1) peptide.…”