Publication: Development of new RNAi therapeutics
Authors
Liu, G. ; Wong-Staal, F. ; Li, Q.X.
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Publisher
Murcia : F. Hernández
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DOI
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info:eu-repo/semantics/article
Description
Abstract
RNAi-mediated gene inactivation has
become a cornerstone of the present day gene function
studies that are the foundation of mechanism and target
based drug discovery and development, which could
potentially shorten the otherwise long process of drug
development. In particular, the coming of age of “RNAi
drug” could provide new promising therapeutics
bypassing traditional approaches. However, there are
technological hurdles need to overcome and the
biological limitations need to consider for achieving
effective therapeutics. Major hurdles include the intrinsic
poor pharmacokinetic property of siRNA and major
biological restrictions include off-target effects,
interferon response and the interference with
endogenous miRNA. Recent innovations in nucleic acid
chemistry, formulations and delivery methods have
gradually rendered it possible to develop effective
RNAi-based therapeutics. Careful design based on the newest RNAi/miRNA biology can also help to minimize
the potential tissue toxicity. If successful with systemic
application, RNAi drug will no doubt revolutionize the
whole drug development process. This review attempts
to describe the progress in this area, including
applications in preclinical models and recent favorable
experience in a number of human trials of local diseases,
along with the discussion on the potential limitations of
RNAi therapeutics.
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