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Long-Term and Real-World Safety and Efficacy of Retroviral Gene Therapy for Adenosine Deaminase Deficiency
Adenosine-deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34+ cell gene therapy (GT) following busulfan reduced-intensity conditioning is a promising therapeutic approach for ADA-SCID, but long-term data are warranted. He...
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| 出版年: | Nat Med |
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| 主要な著者: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2024
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7615698/ https://ncbi.nlm.nih.gov/pubmed/38355973 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-023-02789-4 |
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