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Overcoming Gene-Delivery Hurdles: Physiological Considerations for Nonviral Vectors.


ABSTRACT: With the use of contemporary tools and techniques, it has become possible to more precisely tune the biochemical mechanisms associated with using nonviral vectors for gene delivery. Consequently, nonviral vectors can incorporate numerous vector compositions and types of genetic cargo to develop diverse genetic therapies. Despite these advantages, gene-delivery strategies using nonviral vectors have poorly translated into clinical success due to preclinical experimental design considerations that inadequately predict therapeutic efficacy. Furthermore, the manufacturing and distribution processes are critical considerations for clinical application that should be considered when developing therapeutic platforms. In this review, we evaluate potential avenues towards improving the transition of gene-delivery technologies from in vitro assessment to human clinical therapy.

SUBMITTER: Hill AB 

PROVIDER: S-EPMC5800990 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature

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Overcoming Gene-Delivery Hurdles: Physiological Considerations for Nonviral Vectors.

Hill Andrew B AB   Chen Mingfu M   Chen Chih-Kuang CK   Pfeifer Blaine A BA   Jones Charles H CH  

Trends in biotechnology 20151223 2


With the use of contemporary tools and techniques, it has become possible to more precisely tune the biochemical mechanisms associated with using nonviral vectors for gene delivery. Consequently, nonviral vectors can incorporate numerous vector compositions and types of genetic cargo to develop diverse genetic therapies. Despite these advantages, gene-delivery strategies using nonviral vectors have poorly translated into clinical success due to preclinical experimental design considerations that  ...[more]

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