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- W1991245695 abstract "Cystic fibrosis is a monogenic disorder with significant morbidity and mortality, despite advances in conventional treatment. It is a good candidate for gene therapy and this field has progressed rapidly since the cystic fibrosis transmembrane conductance regulator gene was cloned. We will review the specific questions to address for successful cystic fibrosis gene therapy, such as the extra- and intracellular barriers to airway gene transfer, the target cells and the endpoints to assess efficacy. We will discuss recent advances in viral and nonviral gene transfer agents, delivery techniques and novel strategies to enhance airway gene transfer and expression. Keywords: Cystic fibrosis, Gene therapy, Vectors" @default.
- W1991245695 created "2016-06-24" @default.
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- W1991245695 date "2006-05-01" @default.
- W1991245695 modified "2023-10-01" @default.
- W1991245695 title "Cystic Fibrosis Gene Therapy: Key Questions and Prospects" @default.
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- W1991245695 doi "https://doi.org/10.2174/157339606776894612" @default.
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