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Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia
Gene therapy for hypercholesterolemia offers the potential to sustainably ameliorate disease for life with a single dose. In this study, we demonstrate the combinatorial effects of codon and vector optimization, which significantly improve the efficacy of an adeno-associated virus (AAV) vector in th...
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| Pubblicato in: | Mol Ther Methods Clin Dev |
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| Autori principali: | , , , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society of Gene & Cell Therapy
2021
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8237527/ https://ncbi.nlm.nih.gov/pubmed/34258325 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.04.017 |
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