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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