Gene silencing in the therapy of influenza and other respiratory diseases: Targeting to RNase P by use of External Guide Sequences (EGS).
Respiratory diseases provide an attractive target for gene silencing using small nucleic acids since the respiratory epithelium can be reached by inhalation therapy. Natural surfactant appears to facilitate the uptake and distribution of these types of molecules making aerosolized nucleic acids a possible new class of therapeutics. This article will review the rationale for the use of External Guide Sequence (EGS) in targeting specific mRNA molecules for RNase P-mediated intracellular destruction. Specific destruction of target mRNA results in gene-specific silencing similar to that instigated by siRNA via the RISC complex. The application of EGS molecules specific for influenza genes are discussed as well as the potential for synergy with siRNA. Furthermore, EGS could be adapted to target other respiratory diseases of viral etiology as well as conditions such as asthma.
Dreyfus
David H
DH
Department of Pediatrics, Yale University School of Medicine, New Haven, CT, USA;
Tompkins
S Mark
SM
Fuleihan
Ramsay
R
Ghoda
Lucy Y
LY
Proc Natl Acad Sci U S A. 2004 Jun 8;101(23):8682-6
15173583
Hepatology. 2006 Sep;44(3):527-35
16941688
Nucleic Acids Res. 2006;34(16):4467-76
16945958
Clin Exp Allergy. 2006 Aug;36(8):1049-62
16911361
Int Immunopharmacol. 2004 Aug;4(8):1015-27
15222976
Thorax. 2006 Sep;61(9):809-16
16936237
J Biol Chem. 2002 Aug 16;277(33):30112-20
12050148
Proc Natl Acad Sci U S A. 2004 Jun 15;101(24):9073-8
15184661
RNA. 2006 Jan;12(1):63-72
16301604
Mol Cell. 2007 May 25;26(4):523-37
17531811
JAMA. 2006 Aug 16;296(7):815-26
16905787
AIDS Rev. 2006 Apr-Jun;8(2):60-77
16848274
Nature. 2001 Jan 18;409(6818):363-6
11201747
BioDrugs. 2006;20(1):1-11
16573347
Methods Enzymol. 2005;392:227-41
15644185
Gene Ther. 2007 Jan;14(2):99-107
17167496
Nat Biotechnol. 2000 Jan;18(1):58-61
10625392
Antisense Nucleic Acid Drug Dev. 1998 Oct;8(5):415-26
9826268
Gene. 2003 Aug 14;313:59-69
12957377
Nat Biotechnol. 2003 Jun;21(6):635-7
12754523
Methods Enzymol. 2000;313:442-56
10595372
Tohoku J Exp Med. 2005 Oct;207(2):109-18
16141679
Biochemistry. 2000 Aug 15;39(32):9909-16
10933810
J Biol Chem. 2004 Mar 26;279(13):12542-50
14729660
J HIV Ther. 2005 Dec;10(4):68-71
16519245
J Exp Med. 2001 Jun 4;193(11):1247-60
11390432
Pancreas. 2005 Nov;31(4):373-9
16258373
J Cell Mol Med. 2005 Oct-Dec;9(4):840-53
16364194
Science. 1989 Dec 22;246(4937):1578-84
2480641
J Biol Chem. 1988 Feb 15;263(5):2344-51
3123492
J Biol Chem. 1993 Jul 5;268(19):14514-22
8390996
Nat Genet. 2003 Jul;34(3):263-4
12796781
Cells Tissues Organs. 2004;177(3):132-8
15388987
Am J Physiol Lung Cell Mol Physiol. 2004 Nov;287(5):L1066-70
15234906
Genes Dev. 1989 Apr;3(4):488-99
2470644
Gene Ther. 2001 Dec;8(24):1863-71
11821940
J Immunol. 2004 Sep 15;173(6):4040-9
15356153
J Virol. 2007 Apr;81(7):3428-36
17251292
Nucleic Acids Res. 2006 Jan 23;34(2):575-83
16432261
J Immunol. 2001 Mar 1;166(5):3542-8
11207314
Clin Exp Pharmacol Physiol. 2006 May-Jun;33(5-6):533-40
16700890
Nature. 1996 Dec 12;384(6609):529-34
8955267
Proc Natl Acad Sci U S A. 2004 Feb 17;101(7):1892-7
14769924
Blood. 1998 Dec 15;92(12):4529-38
9845517
Adv Drug Deliv Rev. 2007 Mar 30;59(2-3):124-33
17459519
Cell. 1976 Sep;9(1):101-16
10085
Surgery. 2007 Aug;142(2):262-9
17689694
Proc Natl Acad Sci U S A. 1998 Jun 23;95(13):7327-32
9636148
Nature. 1997 Feb 20;385(6618):721-5
9034188
Expert Opin Investig Drugs. 1997 Sep;6(9):1149-56
15991889
J Mol Biol. 2005 Dec 2;354(3):529-35
16253270
J Biol Chem. 1996 Jun 14;271(24):14533-40
8662854
Virology. 2006 Jan 5;344(1):151-7
16364746
RNA. 2005 Oct;11(10):1588-95
16131590
Bioorg Med Chem Lett. 2004 Oct 4;14(19):4941-4
15341956
Biochemistry. 1992 Dec 8;31(48):12055-61
1280997
Clin Cancer Res. 2004 Jun 1;10(11):3831-8
15173092
J Clin Invest. 2005 Feb;115(2):231-3
15690079
Nucleic Acids Res Suppl. 2001;(1):213-4
12836340
Cell. 1988 Apr 22;53(2):267-72
3359488
FEBS Lett. 2005 Oct 31;579(26):5965-73
16154568
Am Rev Respir Dis. 1976 Feb;113(2):149-53
174464
Curr Top Microbiol Immunol. 2006;311:1-16
17048703
Proc Natl Acad Sci U S A. 2005 Dec 20;102(51):18420-4
16339901
Nucleic Acids Symp Ser. 2000;(44):207-8
12903341
J Allergy Clin Immunol. 2001 Feb;107(2):279-86
11174194
Antiviral Res. 2004 Jan;61(1):49-55
14670593
Biochem Soc Trans. 2007 Aug;35(Pt 4):807-10
17635153
Genet Vaccines Ther. 2007 Feb 01;5:4
17270047
Biochem J. 2004 Jun 15;380(Pt 3):611-6
15096096
J Biol Chem. 1985 May 25;260(10):5942-9
2581945
J Biol Chem. 2002 Mar 1;277(9):6759-62
11741968
Proc Natl Acad Sci U S A. 2003 Feb 18;100(4):1598-602
12552092
Proc Natl Acad Sci U S A. 2002 Mar 19;99(6):3406-11
11904403
J Virol. 2006 Nov;80(21):10579-90
16928753
Pediatr Allergy Immunol. 2002 Oct;13(5):350-6
12431194
J Biol Chem. 1972 Aug 25;247(16):5243-51
4560501
Am J Respir Cell Mol Biol. 2006 Jan;34(1):49-55
16195539
Trends Genet. 2004 Nov;20(11):521-4
15475108
J Virol. 2005 Jun;79(12):7363-70
15919891
J Mol Biol. 2004 Sep 24;342(4):1077-83
15351636