Lentiviral gene therapy for the treatment of ABCA3-mediated surfactant deficiency
Gene therapies are being developed for many rare genetic diseases and provide hope for patients and their families who currently have few treatment options available. Lung surfactant deficiencies comprise a group of rare genetic lung diseases, where babies present at birth with severe respiratory di...
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| フォーマット: | 学位論文 |
| 言語: | English |
| 出版事項: |
2024
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