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2016 ; 82
(3
): 659-72
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Therapeutic nucleic acids: current clinical status
#MMPMID27111518
Sridharan K
; Gogtay NJ
Br J Clin Pharmacol
2016[Sep]; 82
(3
): 659-72
PMID27111518
show ga
Deoxyribonucleic acid (DNA) and ribonucleic acid (RNA) are simple linear polymers
that have been the subject of considerable research in the last two decades and
have now moved into the realm of being stand-alone therapeutic agents. Much of
this has stemmed from the appreciation that they carry out myriad functions that
go beyond mere storage of genetic information and protein synthesis. Therapy with
nucleic acids either uses unmodified DNA or RNA or closely related compounds.
From both a development and regulatory perspective, they fall somewhere between
small molecules and biologics. Several of these compounds are in clinical
development and many have received regulatory approval for human use. This review
addresses therapeutic uses of DNA based on antisense oligonucleotides, DNA
aptamers and gene therapy; and therapeutic uses of RNA including micro RNAs,
short interfering RNAs, ribozymes, RNA decoys and circular RNAs. With their
specificity, functional diversity and limited toxicity, therapeutic nucleic acids
hold enormous promise. However, challenges that need to be addressed include
targeted delivery, mass production at low cost, sustaining efficacy and
minimizing off-target toxicity. Technological developments will hold the key to
this and help accelerate drug approvals in the years to come.
|Aptamers, Nucleotide/therapeutic use
[MESH]
|DNA/*therapeutic use
[MESH]
|Genetic Therapy/methods
[MESH]
|Humans
[MESH]
|MicroRNAs/therapeutic use
[MESH]
|Oligonucleotides, Antisense/therapeutic use
[MESH]