Gene therapy for surfactant protein B deficiency using recombinant AAV vectors
Gene therapies are being developed for rare genetic diseases and offer hope for conditions with few treatment options. Surfactant protein B (SP-B) deficiency is a rare genetic disease of the lung causing severe respiratory distress in neonates and is fatal within the first months of life. Recombinan...
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| Format: | Thesis |
| Sprog: | English |
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2021
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