Gene therapy and infectious diseases

[Jin S Zhang]

Can gene therapy be used to cure the infectious diseases?

 

[Monique]

It may be possible in some cases, but we still need technological advances and many clinical studies before it can be applied to treat diseases.

Here is a paper from 1998: http://www.pubmedcentral.nih.gov/picrender.fcgi?artid=121375&blobtype=pdf And here one from 2005: http://nar.oxfordjournals.org/cgi/reprint/33/1/235

 

[JonMoulton]

Here are some fairly recent preclinical antiviral reports using Morpholino antisense oligos, mostly targeted to viral transcripts.

Moerdyk-Schauwecker M, Stein DA, Eide K, Blouch RE, Bildfell R, Iversen P, Jin L. Inhibition of HSV-1 ocular infection with morpholino oligomers targeting ICP0 and ICP27. Antiviral Res. 2009 Aug 6. [Epub ahead of print]

Gabriel G, Nordmann A, Stein DA, Iversen PL, Klenk HD. Morpholino oligomers targeting the PB1 and NP genes enhance the survival of mice infected with highly pathogenic influenza A H7N7 virus. J Gen Virol. 2008 Apr;89(Pt 4):939-48.

Krähling V, Stein DA, Spiegel M, Weber F, Mühlberger E. SARS-Coronavirus triggers apoptosis via protein kinase R but is resistant to its antiviral activity. J Virol. 2008 Dec 24. [Epub ahead of print]

Lai SH, Stein DA, Guerrero-Plata A, Liao SL, Ivanciuc T, Hong C, Iversen PL, Casola A, Garofalo RP. Inhibition of Respiratory Syncytial Virus Infections With Morpholino Oligomers in Cell Cultures and in Mice. Mol Ther. 2008 Apr 29; [Epub ahead of print]

Lupfer C, Stein DA, Mourich DV, Tepper SE, Iversen PL, Pastey M. Inhibition of influenza A H3N8 virus infections in mice by morpholino oligomers. Arch Virol. 2008 May;153(5):929-37. Epub 2008 Mar 28.

Paessler S, Rijnbrand R, Stein DA, Ni H, Yun NE, Dziuba N, Borisevich V, Seregin A, Ma Y, Blouch R, Iversen PL, Zacks MA. Inhibition of alphavirus infection in cell culture and in mice with antisense morpholino oligomers. Virology. 2008 Jul 5;376(2):357-70. Epub 2008 May 12.


Patel D, Opriessnig T, Stein DA, Halbur PG, Meng XJ, Iversen PL, Zhang YJ. Peptide-conjugated morpholino oligomers inhibit porcine reproductive and respiratory syndrome virus replication. Antiviral Res. 2008 Feb;77(2):95-107. Epub 2007 Oct 4

Sleeman K, Stein DA, Tamin A, Reddish M, Iversen PL, Rota PA. Inhibition of measles virus infections in cell cultures by peptide-conjugated morpholino oligomers. Virus Res. 2008 Dec 4. [Epub ahead of print]

Stein DA, Huang CY, Silengo S, Amantana A, Crumley S, Blouch RE, Iversen PL, Kinney RM. Treatment of AG129 mice with antisense morpholino oligomers increases survival time following challenge with dengue 2 virus. J Antimicrob Chemother. 2008 Sep;62(3):555-65. Epub 2008 Jun 19.

Stein DA, Shi PY. Nucleic acid-based inhibition of flavivirus infections. Front Biosci. 2008 Jan 1;13:1385-95.

Stone JK, Rijnbrand R, Stein DA, Ma Y, Yang Y, Iversen PL, Andino R. A morpholino oligomer targeting highly conserved internal ribosome entry site sequence is able to inhibit multiple species of picornavirus. Antimicrob Agents Chemother. 2008 Jun;52(6):1970-81. Epub 2008 Mar 17.

Zhang B, Dong H, Stein DA, Shi PY. Co-selection of West Nile virus nucleotides that confer resistance to an antisense oligomer while maintaining long-distance RNA/RNA base pairings. Virology. 2008 Dec 5;382(1):98-106. Epub 2008 Oct 7.

Zhang B, Dong H, Stein DA, Iversen PL, Shi PY. West Nile virus genome cyclization and RNA replication require two pairs of long-distance RNA interactions. Virology. 2008 Mar 30;373(1):1-13. Epub 2008 Feb 6.

Zhang YJ, Bonaparte RS, Patel D, Stein DA, Iversen PL. Blockade of viral interleukin-6 expression of Kaposi's sarcoma-associated herpesvirus. Mol Cancer Ther. 2008 Mar;7(3):712-20.

Burrer R, Neuman BW, Ting JPC, Stein DA, Moulton HM, Iversen PL, Kuhn P, Buchmeier MJ. Antiviral effects of antisense morpholino oligomers in murine Coronavirus infection models. J. Virol. 2007 Jun;81(11):5637-48. Epub 2007 Mar 7.

Deas TS, Bennett CJ, Jones SA, Tilgner M, Ren P, Behr MJ, Stein DA, Iversen PL, Kramer LD, Bernard KA, Shi PY. In vitro resistance selection and in vivo efficacy of morpholino oligomers against West Nile virus. Antimicrob Agents Chemother. 2007 Jul;51(7):2470-82. Epub 2007 May 7.

Moulton HM, Fletcher S, Neuman BW, McClorey G, Stein DA, Abes S, Wilton SD, Buchmeier MJ, Lebleu B, Iversen PL. Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine Coronavirus replication in vivo. Biochem Soc Trans. 2007 Aug;35(Pt 4):826-8.

Vagnozzi A, Stein DA, Iversen PL, Rieder E. Inhibition of foot-and-mouth disease virus in cell cultures with antisense Morpholino oligomers. J Virol. 2007 Nov;81(21):11669-80. Epub 2007 Aug 29.

Enterlein S, Warfield KL, Swenson DL, Stein DA, Smith JL, Gamble CS, Kroeker AD, Iversen PL, Bavari S, Muhlberger E. VP35 Knockdown Inhibits Ebola Virus Amplification and Protects against Lethal Infection in Mice. Antimicrob Agents Chemother. 2006 Mar;50(3):984-93.

Warfield KL, Swenson DL, Olinger GG, Nichols DK, Pratt WD, Blouch R, Stein DA, Aman MJ, Iversen PL, Bavari S. Gene-Specific Countermeasures against Ebola Virus Based on Antisense Phosphorodiamidate Morpholino Oligomers. PLoS Pathog. 2006 Jan;2(1):e1. Epub 2006 Jan 13.

Yuan J, Stein DA, Lim T, Qiu D, Coughlin S, Liu Z, Wang Y, Bouch R, Moulton HM, Iversen PL, Yang D. Inhibition of Coxsackievirus B3 in Cell-cultures and in Mice by Peptide-Conjugated Morpholino Oligomers Targeting the IRES. J Virol. 2006 Dec;80(23):11510-9. Epub 2006 Sep 20.