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Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy
Despite significant advances in cystic fibrosis (CF) treatments, a one-time treatment for this life-shortening disease remains elusive. Stable complementation of the disease-causing mutation with a normal copy of the CF transmembrane conductance regulator (CFTR) gene fulfills that goal. Integrating...
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| Publicado no: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7973238/ https://ncbi.nlm.nih.gov/pubmed/33768133 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.02.020 |
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