Klinična študija NS-089/NCNP-02 (exon skipping 44) je pokazala visoko učinkovitost in prisotnost distrofina v celicah pri bolnikih z DMD.

Z terapijo exon skipping 44 je mogoče preskočiti naslednje mutacije v genu Distrofin:

3-43 4-43 5-43 6-43 9-43

10-43 11-43 13-43 14-43 15-43 16-43 17-43 19-43

21-43 23-43 24-43 25-43 26-43 27-43 28-43 29-43

30-43 31-43 32-43 33-43 34-43 35-43 36-43 37-43 38-43 39-43

40-43 41-43 42-43 43 45 45-54 45-56 45-62

45-65 45-68 45-70 45-71 45-72 45-73 45-74

 

https://www.ncnp.go.jp/topics/2022/20220317e.html?fbclid=IwY2xjawI7m-FleHRuA2FlbQIxMAABHYqO7MqMPpGMnzITUO7s7GTCOhW953MFWtjYeP6HroEdryBovAEloUedA_aem_QlSvpGUIspARnxXXyYc3kg

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