Gene therapy strategies for the treatment of chronic viral hepatitis

Bibliography

• WARRIER I, EWENSTEIN BM, KOERPER MA et al.: Factor IX inhibitors and anaphylaxis in hemophilia B. j Pediatr. HeinatoL °rico]. (1997) 19:23–27.
   PubMed Web of Science ®Google Scholar
• HOYER LW: Factor VIII inhibitors. Curr. Opin. HeinatoL (1995) 2:365–371.
   PubMedGoogle Scholar
• ANTONELLI G: Development of neutralizing and binding antibodies to interferon (IFN) in patients undergoing IFN therapy. Antiviral Res. (1994) 24:235–244.
   PubMed Web of Science ®Google Scholar
• RAPPAPORT AM: In Diseases of the Liver Schiff L, Schiff ER (Eds.) JB Lippincott Co., Philadelphia (1987):22.
   Google Scholar
• GUYTON AC: Medical Physiology 6th ed. W.B. Saunders Co., (1981).
   Google Scholar
• CENTERS FOR DISEASE CONTROL AND PREVENTION: Recommendations for the prevention and control of hepatitis C virus (HCV) infection and HCV-related chronic disease. MMWR (1998) 47 (no. RR-19):inclusive.
   Google Scholar
• HOOFNAGLE JH, DI BISCEGLIE AM: The treatment of chronic viral hepatitis. Drug Therapy (1997) 336:347–356.
   Google Scholar
• •Review of currently available therapies for chronic viral hepatitis.
   Google Scholar
• Drug Facts and Comparisons. Olin BR (Ed.), Facts and Comparisons, St. Louis, MO, (1996).
   Google Scholar
• DAVIS GL: Current therapy for chronic hepatitis C. Gastroenterology (2000) 118:5104–5114.
   Google Scholar
• ETO T, TAKAHASHI H: Enhanced inhibition of hepatitis B virus production by asialoglycoprotein receptor-directed interferon. Nature Med. (1999) 5:577–581.
   PubMed Web of Science ®Google Scholar
• ••Specific targeting of IFN-a to the liver toenhance antiviral activity against HBV in an animal model.
   Google Scholar
• TABATA Y, MATSUI Y, UNO K, SOKAWAY, IKADA Y: Simple mixing of IFN with a polysaccharide having high liver affinity enables IFN to target to the liver. I Interferon Cytokine Res. (1999) 19:287–292.
   PubMed Web of Science ®Google Scholar
• •Specific targeting of IFN-a to the liver and demonstration of protein activity.
   Google Scholar
• XI K, TABATA Y, UNO K et a/.: Liver targeting of interferon through pullulan conjugation. Pharin. Res. (1996) 13:1846–1850.
   Google Scholar
• NOISAKRAN S, CAMPBELL IL, CARR DJ: Ectopic expression of DNA encoding IFN-Ot 1 in the cornea protects mice from herpes simplex virus Type 1-induced encephalitis. j InunurioL (1999) 162:4184–4190.
   Google Scholar
• NOISAKRAN SJ, CARR DJ: Therapeutic efficacy of DNA encoding IFN-00 against corneal HSV-1 infection. Curr. Eye Res. (2000) 20:405–4012.
   PubMed Web of Science ®Google Scholar
• •Inhibition of herpes simplex virus by IFN-a gene therapy in corneal tissue.
   Google Scholar
• YEOW WS, LAWSON CM, BEILHARZ IVIW: Antiviral activities of individual murine IFN-a subtypes in vivo: intramuscular injection of IFN expression constructs reduces cytomegalovirus replication. Immunal. (1998) 160:2932–2939.
   PubMed Web of Science ®Google Scholar
• ••Inhibition of CMV replication by IFN-agene therapy in the muscle.
   Google Scholar
• MONAHAN PE, SAMULSKI RJ, TAZELAAR J etal.: Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther. (1998) 5:40–49.
   Google Scholar
• HERZOG RW, YANG EY, COUTO LB et al.: Long-term correction of canine hemophilia B by gene transfer of blood coagulation Factor IX mediated by adeno-associated viral vector. Nature Med. (1999) 5:56–63.
   PubMed Web of Science ®Google Scholar
• AURISICCHIO L, DELMASTRO P, SALUCCI V et al.: Liver-specific -a 2 interferon gene expression results in protection from induced hepatitis. j Viral. (2000) 74:4816–4823.
   PubMed Web of Science ®Google Scholar
• ••Demonstration that liver-specific IFN-agene therapy can provide hepatic protection from acute viral hepatitis, or hepatitis induced by immune system stimulation.
   Google Scholar
• PROTZER U, NASSAL M, CHIANG PW, KIRSCHFINK M, SCHALLER H: Interferon gene transfer by a hepatitis B virus vector efficiently suppresses wild-type virus infection. Proc. Nati Acad. Sci. USA (1999) 96:10818–10823.
   PubMed Web of Science ®Google Scholar
• ••Novel use of recombinant HBV vectors todeliver and express IFN-a gene in the liver. Showed limited ability to inhibit hepatitis virus.
   Google Scholar
• CHIOU HC, LUCAS MA, WU CCN et al.: Long term expression and absence of murine immune response to human interferon after intravenous delivery to the liver by polyplex and formulants. Mol. Ther. (2000) l\(part 2):S126.
   Google Scholar
• GUIDOTTI LG, ANDO K, HOBBS MV et al.: Cytotoxic T lymphocytes inhibit hepatitis B virus gene expression by a noncytolytic mechanism in transgenic mice. Proc. Natl. Acad. Li. USA (1994) 91:3764–3768.
   PubMed Web of Science ®Google Scholar
• •Demonstration of IFN-a-sensitivitiy in a transgenic HBV mouse model.
   Google Scholar
• LARKIN J, CLAYTON M, SUN B et al.: Hepatitis B virus transgenic mouse model of chronic liver disease. Nature Med. (1999) 5:907–912.
   PubMed Web of Science ®Google Scholar
• •Transgenic HBV mouse model that can be induced to develop a chronic hepatitis-like state.
   Google Scholar
• GUIDOTTI LG, CHISARI FV: Cytokine-mediated control of viral infections. Virology (2000) 273:221–227.
   PubMed Web of Science ®Google Scholar
• •Review of cytokine-mediated mechanisms for inhibition of viral hepatitis.
   Google Scholar
• HUARD J, LOCHMULLER H, ACSADI G, JANI A, MASSIE B, KARPATI G: The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther. (1995) 2:107–115.
   PubMed Web of Science ®Google Scholar
• BENIHOUD K, YEH P, PERRICAUDET M: Adenovirus vectors for gene delivery. Cum: Opin. Biatechnol. (1999) 10:440–447.
   PubMed Web of Science ®Google Scholar
• MONAHAN PE, SAMULSKI RJ: AAV vectors: is clinical success on the horizon? Gene Ther. (2000) 7:24–30.
   Google Scholar
• ILAN Y, SAITO H, THUMMALA NR, CHOWDHURY NR: Adenovirus-mediated gene therapy of liver diseases. Sea-1M Liver Dis. (1999) 19:49–59.
   Google Scholar
• ••Review of various methods to circumventimmune response to recombinant viral vectors commonly used in gene therapy.
   Google Scholar
• BREINER KM, URBAN S, GLASS B, SCHALLER H: Envelope protein-mediated down-regulation of hepatitis B virus receptor in infected hepatocytes. j Viral. (2001) 75:143–150.
   PubMed Web of Science ®Google Scholar
• •Study which showed that a primary infection by HBV inhibits subsequent superinfection by the same virus.
   Google Scholar
• LIAW YF: Role of hepatitis C virus in dual and triple hepatitis virus infection.Hepatology (1995) 22:1101–1108.
   PubMed Web of Science ®Google Scholar
• •Report of suppression of HBV by HCV in a mixed infection in patients.
   Google Scholar
• LIAW YE YEH CT, TSAI SL: Impact of acute hepatitis B virus superinfection on chronic hepatitis C virus infection. Am. Gastroenteral. (2000) 95:2978–2980.
   PubMed Web of Science ®Google Scholar
• CHEN Si SU H, YEE JK: Repression of liver-specific hepatitis B virus enhancer 2 activity by adenovirus ElA proteins. Viral. (1992) 66:7452–7460.
   PubMed Web of Science ®Google Scholar
• SCHAACK J, MAGUIRE HE SIDDIQUI A: Hepatitis B virus X protein partially substitutes for ElA transcriptional function during adenovirus infection. Virology (1996) 216:425–430.
   PubMed Web of Science ®Google Scholar
• •Study reports on interactions between HBV and adenovirus infections and the ability, in some specific cases, of a viral function from one species being able to complement a missing function from another.
   Google Scholar
• PASTORE G, SANTANTONIO T, MONNO L, MILELLA M, LUCHENA N, ANGARANO G: Effects of HIV superinfection on HBV replication in a chronic HBsAg carrier with liver disease. Hepatal. (1988) 7:164–168.
   PubMed Web of Science ®Google Scholar
• SCHLEHOFER JR, ZUR HAUSEN H: Adenovirus infection induces amplification of persistent viral DNA sequences (simian virus 40, hepatitis B virus, bovine papillomavirus) in human and rodent cells. Virus Res. (1990) 17:53–60.
   PubMed Web of Science ®Google Scholar
• ZEUZEM S, FEINMAN SV, RASENACK J et al.: Peginterferon alfa-2a in patients with chronic hepatitis C. N Engl. I Med. (2000) 343:1666–1672.
   PubMed Web of Science ®Google Scholar
• •Enhancement of IFN-a activity by increasing its circulatory half-life by modification with polyethylene glycol.
   Google Scholar
• HEATHCOTE EJ, SHIFFMAN ML, COOKSLEY WG et al.: Peginterferon alfa-2a in patients with chronic hepatitis C and cirrhosis. N Engl. J Med. (2000) 343:1673–1680.
   PubMed Web of Science ®Google Scholar
• •Enhancement of IFN-a activity by increasing its circulatory half-life by modification with polyethylene glycol.
   Google Scholar
• GLUE P, FANG JW, ROUZIER-PANIS R etal.: Pegylated interferon-al2b: pharmacokinetics, pharmacodynamics, safety, and preliminary efficacy data. Hepatitis C Intervention Therapy Group. Chu. Pharmacal. Ther. (2000) 68:556–567.
   Google Scholar
• WANG L, TAKABE K, BIDLINGMAIER SM, ILL CR, VERMA IM: Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc. Natl. Acad. Sci. USA (1999) 96:3906–3910.
   PubMed Web of Science ®Google Scholar
• SCAGLIONI PP, MELEGARI M, WANDS JR: Characterization of hepatitis B virus core mutants that inhibit viral replication. Virology (1994) 205:112–120.
   PubMed Web of Science ®Google Scholar
• •One of the first papers characterising dominant negative mutant core proteins and their potential as anti-HBV agents.
   Google Scholar
• SCAGLIONI P, MELEGARI M, TAKAHASHI M, CHOWDHURY JR, WANDS J: Use of dominant negative mutants of the hepadnaviral core protein as antiviral agents. Hepatology (1996) 24:1010–1017.
   PubMed Web of Science ®Google Scholar
• VON WEIZSACKER F, WIELAND S, BLUM HE: Inhibition of viral replication by genetically engineered mutants of the duck hepatitis B virus core protein. Hepatology (1996) 24:294–299.
   PubMed Web of Science ®Google Scholar
• ••Demonstration that expression of amodified core protein, exhibiting a dominant negative phenotype, can inhibit hepadnaviral virion production by inhibition of nucleocapsid formation. The 637 authors propose that expression of this type of mutant protein could be employed as an anti-HBV gene therapy.
   Google Scholar
• LARAS A, KOSKINAS J, AVGIDIS KHADZIYANNIS SJ: Incidence and clinical significance of hepatitis B virus precore gene translation initiation mutations in e antigen-negative patients. Viral Hepatitis (1998) 5:241–248.
   PubMed Web of Science ®Google Scholar
• LINDH M, HORAL P, DHILLON AI NORKRANS G: Hepatitis B virus DNA levels, precore mutations, genotypes and histological activity in chronic hepatitis B. Viral Hepatitis (2000) 7:258–267.
   PubMed Web of Science ®Google Scholar
• BUCKWOLD VE, XU Z, CHEN M, YEN TS, OU JH: Effects of a naturally occurring mutation in the hepatitis B virus basal core promoter on precore gene expression and viral replication. j Viral. (1996) 70:5845–5851.
   PubMed Web of Science ®Google Scholar
• •Provides good understanding of the mechanisms involved in suppression of viral replication by HBV precore protein.
   Google Scholar
• SCAGLIONI PP, MELEGARI M, WANDS JR: Posttranscriptional regulation of hepatitis B virus replication by the precore protein. I Viral. (1997) 71:345–353.
   Google Scholar
• ••Specific engineering of the HBV precoregene to produce a non-secreting protein could significantly increase the potency of a gene therapy based upon expression of the precore protein for the inhibition of HBV virion production.
   Google Scholar
• GUIDOTTI LG, MATZKE B, PASQUINELLI C, SHOENBERGER JM, ROGLER CE, CHISARI FV: The hepatitis B virus (HBV) precore protein inhibits HBV replication in transgenic mice. Viral. (1996) 70:7056–7061.
   PubMed Web of Science ®Google Scholar
• CHISARI FV: Hepatitis B virus transgenic mice: insights into the virus and the disease. Hepatology(1995) 22:1316–1325.
   PubMed Web of Science ®Google Scholar
• RUIZ J, QIAN C, DROZDZIK M, PRIETO J: Gene therapy of viral hepatitis and hepatocellular carcinoma. j Viral Hepatitis (1999) 6:17–34.
   PubMed Web of Science ®Google Scholar
• ••Broad review of various strategies to treatviral hepatitis by gene therapy, including antisense, gene vaccination and immunotherapeutic strategies.
   Google Scholar
• VON WEIZSACKER F, WIELAND S, KOCK J et al.: Gene therapy for chronic viral hepatitis: ribozymes, antisense oligonucleotides, and dominant negative mutants. Hepatology (1997) 26:251–255.
   PubMed Web of Science ®Google Scholar
• CASELMANN WH, EISENHARDT S, ALT M: Synthetic antisense oligodeoxynucleotides as potential drugs against hepatitis C. Intervirology (1997) 40:394–399.
   PubMed Web of Science ®Google Scholar
• •Review of antisense based anti-hepatitis virus strategies.
   Google Scholar
• MONTEITH DK, LEVIN AA: Synthetic oligonucleotides: the development of antisense therapeutics. Taxical. Patna. (1999) 27:8–13.
   PubMed Web of Science ®Google Scholar
• •Summary of pharmacokirtetics and toxicities of phosphorothioate oligonucleotides.
   Google Scholar
• SOMIA N, VERMA IM: Gene therapy: trials and tribulations. Nature Rev (2000) 1:91–99.
   Web of Science ®Google Scholar
• HAN S, MAHATO RI, SUNG YK, KIM SW: Development of biomaterials for gene therapy. Mai Ther. (2000) 2:302–317.
   PubMed Web of Science ®Google Scholar
• ••Extensive review of various polymer-basedsystems currently being developed for gene delivery.
   Google Scholar
• LUO D, SALTZMAN WM: Synthetic DNA delivery systems. Nature Biatechnol. (2000) 18:33–37.
   PubMed Web of Science ®Google Scholar
• •Review of non-viral gene delivery systems and some of the main obstacles impeding their development.
   Google Scholar
• DE SMEDT SC, DEMEESTER J, HENNINK WE: Cationic polymer based gene delivery systems. Pharmaceut. Res. (2000) 17:113–126.
   PubMed Web of Science ®Google Scholar
• SMITH LC, DUGUID J, WADHWA MS etal.: Synthetic peptide-based DNA complexes for nonviral gene delivery. Adv. Drug Deify. Rev (1998) 30:115–131.
   Google Scholar
• BOUSSIF 0, LEZOUALC'H E ZANTA MA et al.: A versatile vector for gene and oligonucleotide transfer into cells in culture and in viva: polyethylenimine. Proc. Natl. Acad. Sd. USA (1995) 92:7297–7301.
   Google Scholar
• •One of the initial reports on the use of PEI as a polymeric DNA delivery system.
   Google Scholar
• KABANOV AV, KABANOV VA: Interpolyelectrolyte and block ionomer complexes for gene delivery: physico-chemical aspects. Adv. Drug Deify. Rev (1998) 30:49–60.
   PubMed Web of Science ®Google Scholar
• PLANK C, TANG MX, WOLFE AR, SZOKA FC JR.: Branched cationic peptides for gene delivery: role of type and number of cationic residues in formation and in vitro activity of DNA polyplexes. Hum. Gene Ther. (1999) 10:319–332.
   Google Scholar
• COLLARD WT, YANG Y, KWOK KY, PARK Y, RICE KG: Biodistribution, metabolism, and in viva gene expression of low molecular weight glycopeptide polyethylene glycol peptide DNA co-condensates. j Pharm. Li. (2000) 89:499–512.
   Web of Science ®Google Scholar
• MACLAUGHLIN FC, MUMPER RJ, WANG J et al.: Chitosan and depolymerized chitosan oligomers as condensing carriers for in viva plasmid delivery. I Control Release (1998) 56:259–272.
   PubMed Web of Science ®Google Scholar
• KUKOWSKA-LATALLO JF, BIELINSKA AU, JOHNSON J, SPINDLER R, TOMALIA DA, BAKER JR: Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers. Proc. Natl. Acad. Sci. USA (1996) 93:4897–4902.
   PubMed Web of Science ®Google Scholar
• ZUIDAM NJ, POSTHUMA G, DE VRIES ET, CROMMELIN DJ, HENNINK WE, STORM G: Effects of physicochemical characteristics of poly(2-(dimethylamino)ethyl methacrylate)-based polyplexes on cellular association and internalization. I Drug Target (2000) 8:51–66.
   PubMed Web of Science ®Google Scholar
• OUPICKY D, KONAK C, ULBRICH K, WOLFERT MA, SEYMOUR LW: DNA delivery systems based on complexes of DNA with synthetic polycations and their copolymers. I Controlled Release (2000) 65:149–171.
   PubMed Web of Science ®Google Scholar
• LOLLO CP, BANASZCZYK MG, CHIOU HC: Obstacles and advances in non-viral gene delivery. Curr. Opin. Mai Ther. (2000) 2:136–142.
   PubMed Web of Science ®Google Scholar
• •Review of some of the current obstacles in polymer-based gene therapy and potential methods of overcoming them.
   Google Scholar
• MIR LM, BUREAU MF, GEHL J et al.: High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc. Natl. Acad. Sci. USA (1999) 96:4262–7.
   PubMed Web of Science ®Google Scholar
• AIHARA H, MIYAZAKI J: Gene transfer into muscle by electroporation in vivo. Nature Biatechnol. (1998) 16:867–870.
   PubMed Web of Science ®Google Scholar
• LIU F, SONG YK, LIU D: Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. (1999) 6:1258–1266.
   PubMed Web of Science ®Google Scholar
• ••One of the initial reports demonstratingthe use of hydrostatic pressure in vivo to achieve gene delivery and expression in the liver.
   Google Scholar
• ZHANG G, BUDKER V, WOLFF JA: High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum. Gene Ther. (1999) 10:1735–1737.
   PubMed Web of Science ®Google Scholar
• ••One of the initial reports demonstrating the use of hydrostatic pressure in vivo to achieve gene delivery and expression in the liver.
   Google Scholar
• BUDKER V, ZHANG G, DANKO I, WILLIAMS P, WOLFF J: The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. (1998) 5:272–276.
   PubMed Web of Science ®Google Scholar
• LAWRIE A, BRISKEN AF, FRANCIS SE et al.: Ultrasound enhances reporter gene expression after transfection of vascular cells in vitro. Circulation (1999) 99:2617–2620.
   PubMed Web of Science ®Google Scholar
• VON DER LEYEN HE, BRAUN-DULLAEUS R, MANN MJ, ZHANG L, NIEBAUER J, DZAU VJ: A pressure-mediated nonviral method for efficient arterial gene and oligonucleotide transfer. Hum. Gene Ther. (1999) 10:2355–2364.
   PubMed Web of Science ®Google Scholar
• ••Demonstration that the use of hydrostaticpressure in vivo can achieve gene delivery to, and expression in smooth muscle.
   Google Scholar
• KURIYAMA N, KURIYAMA H, JULIN CM, LAMBORN K, ISRAEL MA: Pretreatment with protease is a useful experimental strategy for enhancing adenovirus-mediated cancer gene therapy. Hum. Gene Ther. (2000) 11:2219–2230.
   PubMed Web of Science ®Google Scholar
• MAILLARD L, ZIOL M, TAHLIL 0 et al: Pre-treatment with elastase improves the efficiency of percutaneous adenovirus-mediated gene transfer to the arterial media. Gene Ther. (1998) 5:1023–1030.
   PubMed Web of Science ®Google Scholar
• LEGENDRE JY, TRZECIAK A, BOHRMANN B, DEUSCHLE U, KITAS E, SUPERSAXO A: Dioleoylmelittin as a novel serum-insensitive reagent for efficient transfection of mammalian cells. Bioconjug. Chem. (1997) 8:57–63.
   PubMed Web of Science ®Google Scholar
• WAGNER E: Application of membrane-active peptides for nonviral gene delivery. Adv. Drug Deify. Rev (1999) 38:279–289.
   PubMed Web of Science ®Google Scholar
• DANKO I, FRITZ JD, PAO S, HOGAN K, LATENDRESSE JS, WOLFF JA: Pharmacological enhancement of in vivo foreign gene expression in muscle. Gene Ther. (1994) 1:114–121.
   PubMed Web of Science ®Google Scholar
• CROYLE MA, ROESSLER BJ, HSU CI SUN R, AMIDON GL: Beta cyclodextrins enhance adenoviral-mediated gene delivery to the intestine. Pharmaceut. Res. (1998) 15:1348–1355.
   PubMed Web of Science ®Google Scholar
• FREEMAN DJ, NIVEN RW: The influence of sodium glycocholate and other additives on the in vivo transfection of plasmid DNA in the lungs. Pharmaceut. Res. (1996) 13:202–209.
   PubMed Web of Science ®Google Scholar
• ROSS GF, MORRIS RE, CIRAOLO G et al.: Surfactant protein A-polylysine conjugates for delivery of DNA to airway cells in culture. Hum. Gene Ther. (1995) 6:31–40.
   PubMed Web of Science ®Google Scholar
• MALIK AH, LEE WM: Chronic hepatitis B virus infection: treatment strategies for the next millennium. Ann. Intern. Med. (2000) 132:723–731.
   PubMed Web of Science ®Google Scholar