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Table 5 Summary of gene therapy in-vivo results

From: Research, diagnosis and education in inborn errors of metabolism in Colombia: 20 years’ experience from a reference center

Tissue AAV-GALNS AAV-GALNS
AAV-SUMF1
Bone-tagged
AAV-GALNS
Plasma 8.5 19.4 NA
Liver 21.9 36.6 24.3
Spleen 4.5 5.4 3.2
Kidney 3.2 3.1 1.8
Lung 4.2 4.1 3.0
Heart 6.5 30.6 61.8
Brain 4.0 9.1 35.8
Bone marrow 2.0 10.4 13.8
Bone (leg) 0.2 33.3 41.9
  1. Results are expressed as percentage of wild-type levels. NA not assayed