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Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID
Adenosine deaminase (ADA) deficiency is an inborn error of metabolism affecting multiple systems and causing severe combined immunodeficiency. We tested intravenous administration of recombinant adeno-associated virus (AAV) 2/8-ADA vector in ADA-deficient neonate and adult mice or as part of a bimod...
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| Publié dans: | Mol Ther Methods Clin Dev |
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| Auteurs principaux: | , , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society of Gene & Cell Therapy
2021
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7940710/ https://ncbi.nlm.nih.gov/pubmed/33738330 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.02.007 |
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