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- W1999723571 abstract "Recent studies have shed light on a number of important obstacles to safe and effective gene transfer to the respiratory tract with recombinant AAV vectors. Among these are blocks at the level of receptor binding and internalizations, evasion of proteasomal degradation, inefficiency of nuclear entry, and nuclear factors that inhibit the conversion of rAAV genomes into active double-stranded DNA form. Other important issues have been the size constraints of the vector, the lack of retention of episomal forms of the vector genome, and immune responses which may limit the efficiency of repeated doses of rAAV. Each of these potential obstacles has been addressed with new vector designs. In addition, the availability of an abundance of novel rAAV serotypes, each with its own receptor tropism, has expanded the range of possibilities for long-term success of gene therapy in the respiratory tract. Keywords: cystic fibrosis (cf), alpha antitrypsin(aat), raav-mediated gene therapy, capsid mutants, proteasomal degradation, xenografts, tyrosine kinase inihbitors, split-intron vector" @default.
- W1999723571 created "2016-06-24" @default.
- W1999723571 creator A5010005871 @default.
- W1999723571 date "2005-06-01" @default.
- W1999723571 modified "2023-10-02" @default.
- W1999723571 title "Recent Developments in Recombinant AAV-Mediated Gene Therapy for Lung Diseases" @default.
- W1999723571 doi "https://doi.org/10.2174/1566523054064986" @default.
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