N-substituted acetonitrile inhibitors of cathepsin L

Bibliography

• DAVIS PB, DRUMM M, KONSTAN MW: Cystic fibrosis.. J. Respir. Grit. Care Med. (1996) 154:1229–1256.
   PubMed Web of Science ®Google Scholar
• ••An excellent review on all aspects of CF.
   Google Scholar
• WINE JJ: The genesis of cystic fibrosis lung disease. J.. Invest. (1999) 103 :309–312.
   Google Scholar
• ••Review highlighting the conflicting hypotheses on lungdisease.
   Google Scholar
• ALTON EW, GEDDES DM, GILL DR et al.: Towards gene for cystic fibrosis: a clinical progress report. Gene Ther. (1998) 5:291–292.
   PubMed Web of Science ®Google Scholar
• ••Concise summary of CFGT trials and their findings.
   Google Scholar
• KITSON C, ANGEL B, JUDD D et al: The extra- and barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther. (1999) 6:534–546.
   PubMed Web of Science ®Google Scholar
• BOUCHER RC: Status of gene therapy for cystic fibrosis lung disease. J Chn. Invest. (1999) 103:441–445.
   PubMed Web of Science ®Google Scholar
• •Thoughtful review of CFGT and its future prospects.
   Google Scholar
• BUDKER V, ZHANG G, KNECHTLE S, WOLFF JA: Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther. (1996) 3:593–598.
   PubMed Web of Science ®Google Scholar
• PORTEOUS DJ, DORIN JR, MCLACHLAN G et al: Evidence safety and efficacy of DOTAP cationic liposome mediated CFI"? gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. (1997) 4:210–218.
   PubMed Web of Science ®Google Scholar
• ••A Phase I trial showing gene transfer to the nasal epitheliumusing DOTAP/pCMV-CFTR complex.
   Google Scholar
• ALTON EW, STERN M, FARLEY R et al: Cationic lipid- CFIR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet (1999) 353:947–954.
   PubMed Web of Science ®Google Scholar
• ••A comprehensive trial of CFGT using Genzyme's lipid GL-67in a formulation consisting of GL-67/DOPE/DMPE-PEG5000. Evidence for gene transfer was obtained using a number of assays. Importantly, the treatment resulted in mild, transient respiratory symptoms that are almost certainly attributable to the methylation-dependent innate immune defence system.
   Google Scholar
• HYDE SC, SOUTHERN KW, GILEADI U et al: Repeat of DNA/liposomes to the nasal epithe-lium of patients with cystic fibrosis. Gene Ther. (2000) 7:1156–1165.
   PubMed Web of Science ®Google Scholar
• ••A repeat administration trial of CFGT to the nose usingDC-Chol/DOPE. No evidence of inflammation, toxicity or an immune response towards DNA/liposome complex or expressed CFTR was seen: compare with the lung trial of Alton et al. (2000) in which there was transient inflammation associated with gene delivery.
   Google Scholar
• CHESNOY S, HUANG L: Structure and function oflipid-DNA complexes for gene delivery. Ann. Rev. Biophys. Biomol. Struct. (2000) 29:27–47.
   PubMedGoogle Scholar
• ZELPHATI O, HANG X, HOBART P, FELGNER PL: Genechemistry: functionally and conformationally intact fluorescent plasmid DNA. Hum. Gene Ther. (1999) 10:15–24.
   PubMed Web of Science ®Google Scholar
• CHENG SH, SCHEULE RK: Airway delivery of cationiclipid: DNA complexes for cystic fibrosis. Adv. Drug. Deliv. Rev. (1998) 30:173–184.
   PubMed Web of Science ®Google Scholar
• ZABNER J, CHENG SH, MEEKER D et al.: Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. J. Clin. Invest. (1997) 100:1529–1537.
   PubMed Web of Science ®Google Scholar
• ••A nasal trial of CFGT using a GL-67:DOPE formulation. Genetransfer was detected in the treated nostrils and partial electrophysiological correction observed. Intriguingly, DNA alone was also found to be taken up and expressed at a measurable rate.
   Google Scholar
• JENKINS RG, HERRICK SE, MENG QH et al: An integrin-targeted non-viral vector for pulmonary gene therapy. Gene Ther. (2000) 7:393–400.
   PubMed Web of Science ®Google Scholar
• HARVIE P, WONG FM, BALLY MB: Characterization oflipid DNA interactions. I. Destabilization of bound lipids and DNA dissociation. Biophys. J. (1998) 75:1040–1051.
   Google Scholar
• BLESSING T, REMY JS, BEHR JP: Monomolecular collapseof plasmid DNA into stable virus-like particles. Proc. Natl. Acad. Sci. USA (1998) 95:1427–1431.
   PubMed Web of Science ®Google Scholar
• STERNBERG B, HONG K, ZHENG W, PAPAHADJO-POULOS D: Ultrastructur al characterization of cationic liposome-DNA complexes showing enhanced stability in serum and high transfection activity in vivo. Biochim. Biophys. Acta. (1998) 1375:23–35.
   PubMed Web of Science ®Google Scholar
• LIN W, GARNETT MC, DAVIES MC et al: Preparation ofsurface-modified albumin nanospheres. Biomaterials (1997) 18:559–565.
   PubMed Web of Science ®Google Scholar
• TRUONG-LE VL, WALSH SM, SCHWEIBERT E et al: Genetransfer by DNA-gelatin n an o sph er es. Arch. Biochem. Biophys. (1999) 361 :47–56.
   PubMed Web of Science ®Google Scholar
• ILLUM L: Chitosan and its use as a pharmaceutical excipient. Pharm. Res. (1998) 15:1326–1331.
   PubMed Web of Science ®Google Scholar
• AU HC, MASCARELLO JT, SCHEFFLER IE: Targeted integration of a dominant neo(R) marker into a 2- to 3-Mb human minichromosome and transfer between cells. Cytogenet. Cell. Genet. (1999) 86:194–203.
   PubMedGoogle Scholar
• TELENIUS H, SZELES A, KERESO J et al.: Stability of a functional murine satellite DNA-based artificial chromosome across mammalian species. Chromosome Res. (1999) 7:3–7.
   PubMedGoogle Scholar
• CSONKA E, CSERPAN I, FODOR K et al.: Novel generation of human satellite DNA-based artificial chromosomes in mammalian cells. J. Cell Sci. (2000) 113:3207–3216.
   PubMed Web of Science ®Google Scholar
• CRYSTAL RG, MCELVANEY NG, ROSENFELD MA et al.:Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat. Genet. (1994) 8:42–51.
   PubMed Web of Science ®Google Scholar
• CRYSTAL RG, JAFFE A, BRODY S et al.: APhase 1 study, in cystic fibrosis patients, of the safety, toxicity and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung. Hum. Gene Ther. (1995) 6:643–666.
   PubMed Web of Science ®Google Scholar
• ••A pioneering lung trial of adenoviral CFGT.
   Google Scholar
• HARVEY BG, LEOPOLD PL, HACKETT NR et al.: Airway CFIR mRNA expression in cystic fibrosis patients after repetitive administration of a recombi-nant adenovirus. j Clin. Invest (1999) 104:1245–1255.
   PubMed Web of Science ®Google Scholar
• ••A milestone study that revealed the decay of transgeneexpression with repeat dosing of recombinant CFTR adenovirus.
   Google Scholar
• KAPLAN JM, ARMENTANO D, SCARIA A et al.: Novel role E4 region genes in protection of adenovirus from lysis by cytotoxic T lymphocytes. j Vim/ (1999) 73:4489–4492.
   Google Scholar
• MORRAL N, O'NEAL W, RICE K et al.: Administration ofhelper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver- directed gene transfer in baboons. Proc. Natl. Acad. Sci. USA (1999) 96:12816–12821.
   Google Scholar
• HOFMANN C, LOSER P, CICHON G, ARNOLD W, BOTHGW, STRAUSS M: Ovine adenovirus vectors overcome preexisting humoral immunity against human adenoviruses in vivo. J. Viro/. (1999) 73:6930–6936.
   PubMedGoogle Scholar
• ZABNER J, FREIMUTH P, PUGA A, FABREGA A, WELSH MJ: of high affinity fiber receptor activity explains the resistance of ciliated airway epithelia to adenovirus infection. j Clin. Invest (1997) 100:1144–1149.
   PubMedGoogle Scholar
• ••Demonstrates that receptor localisation away from theapical surface prevents efficient adenoviral infection.
   Google Scholar
• WALTERS RW, GRUNST T, BERGELSON JM, FINBERG RW, WELSH MJ, ZABNER J: Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia. J. Biol. Chem. (1999) 274:10219–10226.
   PubMed Web of Science ®Google Scholar
• KASONO K, BLACKWELL JL, DOUGLAS JT et al.: Selectivegene delivery to head and neck cancer cells via an integrin targeted adenoviral vector. Clin. Cancer. Res. (1999) 5:2571–2579.
   PubMed Web of Science ®Google Scholar
• REYNOLDS P, DMITRIEV I, CURIEL D: Insertion of anRGD motif into the HI oop of adenovirus fiber protein alters the distribution of transgene expression of the systemically administered vector. Gene Ther. (1999) 6:1336–1339.
   PubMed Web of Science ®Google Scholar
• ZABNER J, CHILLON M, GRUNST T et al.: A chimeric Type2 adenovirus vector with a Type 17 fiber enhances gene transfer to human airway epithelia. j Vim/ (1999) 73:8689–8695.
   Google Scholar
• ROMANCZUK H, GALER CE, ZABNER J, BARSOMIAN G, WADSWORTH SC, O'RIORDAN CR: Modification of an adenoviral vector with biologically selected peptides: a novel strategy for gene delivery to cells of choice. Hum. Gene Ther. (1999) 10:2615–2626.
   PubMed Web of Science ®Google Scholar
• FASBENDER A, ZABNER J, CHILLON M et al.: Complexes of adenovirus with polycationic polymers and cationic lipids increase the efficiency of gene transfer in vitro and in vivo" Biol. Chem. (1997) 272:6479–6489.
   Google Scholar
• CHILLON M, LEE JH, FASBENDER A, WELSH MJ: Adenovirus complexed with polyethylene glycol and cationic lipid is shielded from neutralizing antibodies in vitro. Gene Ther. (1998) 5:995–1002.
   PubMed Web of Science ®Google Scholar
• FASBENDER A, LEE JH, WALTERS RW, MONINGER TO, ZABNER J, WELSH MJ: Incorporation of adenovirus in calcium phosphate precipitates enhances gene transfer to airway epithelia in vitro and in vivo. J. Clin. Invest. (1998) 102:184–193.
   PubMedGoogle Scholar
• WU N, ATAAI MM: Production of viral vectors for gene therapy applications. Curr. Opin. Biotechnol (2000) 11:205–208.
   PubMedGoogle Scholar
• •A survey of methods of making viral vectors to GT scale.
   Google Scholar
• FANG B, KOCH P, ROTH JA: Diminishing adenovirus gene expression and viral replication by promoter replacement. j Vim/ (1997) 71:4798–4803.
   Google Scholar
• ZHANG L, WANG D, FISCHER H et al.: Efficient expres-sion of CFIR function with adeno-associated virus vectors that carry shortened CFIR genes. Proc. Nad Acad. Sci. USA (1998) 95:10158–10163.
   PubMedGoogle Scholar
• WAGNER JA, MESSNER AH, MORAN ML et al.: Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis tr an sm embr an e regulator (AAV-CFIR) in the cystic fibrosis maxillary sinus. Laryngoscope (1999) 109:266–274.
   PubMed Web of Science ®Google Scholar
• ENGELSTADTER M, BOBKOVA M, BAIER M et al.: Targeting human T cells by retroviral vectors displaying antibody domains selected from a phage display library. Hum. Gene Ther. (2000) 11:293–303.
   PubMed Web of Science ®Google Scholar
• JIANG A, CHU TH, NOCKEN F, CICHUTEK K, DORNBURG R: Cell-type-specific gene transfer into human cells with retroviral vectors that display single-chain antibodies. j Vim/. (1998) 72:10148–10156.
   Google Scholar
• NALDINI L, BLOMER U, GALLAY P et al.: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science (1996) 272:263–267.
   PubMed Web of Science ®Google Scholar
• BLOMER U, NALDINI L, KAFRI T, TRONO D, VERMA IM,GAGE FH: Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector./ Vim/ (1997) 71:6641–6649.
   PubMed Web of Science ®Google Scholar
• GOLDMAN MJ, LEE PS, YANG JS, WILSON JM: lentiviral vectors for gene therapy of cystic fibrosis. Hum. Gene Ther. (1997) 8:2261–2268.
   PubMed Web of Science ®Google Scholar
• GOLDMAN MJ, ANDERSON GM, STOLZENBERG ED, KARI UP, ZASLOFF M, WILSON JM: Human 13-defensin-1 is a salt-sensitive antibiotic in lung that is inactivated in cystic fibrosis. Cell (1997) 88:553–560.
   PubMed Web of Science ®Google Scholar
• JOHNSON LG, OLSEN JC, NALDINI L, BOUCHER RC: Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in viva Gene Ther. (2000) 7:568–574.
   Google Scholar
• WANG G, SLEPUSHKIN V, ZABNER J et al.: Felineimmunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect. J. Clin. Invest. (1999) 104:R55–62.
   PubMed Web of Science ®Google Scholar
• LIN X: Construction of new retroviral producer cells from adenoviral and retroviral vectors. Gene Ther. (1998) 5:1251–1258.
   PubMedGoogle Scholar
• FANG B, KOCH P, BOUVET M, JI L, ROTH JA: Apackaging system for 5V40 vectors without viral coding sequences. Anal. Biochem. (1997) 254:139–143.
   PubMedGoogle Scholar
• BOYCE FM, BUCHER NL: Baculovirus-mediated gene transfer into mammalian cells. Proc. Natl. Acad. Sci. USA (1996) 93:2348–2352.
   PubMed Web of Science ®Google Scholar
• BARSOUM J, BROWN R, MCKEE M, BOYCE FM: Efficient transduction of mammalian cells by a recombinant baculovirus having the vesicular stomatitis virus G glycoprotein. Hum. Gene Ther. (1997) 8:2011–2018.
   PubMedGoogle Scholar
• SENA-ESTEVES M, SAEKI Y, FRAEFEL C, BREAKEFIELD XO:-1 amplicon vectors - simplicity and versatility. Mol. Ther. (2000) 2:9–15.
   PubMed Web of Science ®Google Scholar
• COSTANTINI LC, JACOBY DR, WANG S, FRAEFEL C, BREAKEFIELD XO, ISACSON 0: Gene transfer to the nigrostriatal system by hybrid herpes simplex virus/adeno-associated virus amplicon vectors. Hum. Gene Ther. (1999) 10:2481–2494.
   PubMed Web of Science ®Google Scholar
• PAILLARD F: Promoter attenuation in gene therapy: causes and remedies. Hum. Gene Ther. (19 9 7) 8:2009-2010.
   Google Scholar
• •An editorial drawing attention to the problems of viral promoter attenuation and how they may be addressed.
   Google Scholar
• CHOW YH, O'BRODOVICH H, PLUMB J et al.: Develop-ment of an epithelium-specific expression cassette with human DNA regulatory elements for transgene expression in lung airways. Proc. Natl. Acad. ScL USA (1997) 94:14695–14700.
   Google Scholar
• SMITH DJ, NUTHALL HN, MAJETTI ME, HARRIS A: Multiple potential intragenic regulatory elements in the CEIR gene. Genomics (2000) 64:90–96.
   PubMed Web of Science ®Google Scholar
• GOSSEN M, FREUNDLIEB S, BENDER G, MULLER G, HILLEN W, BUJARD H: Transcriptional activation by tetracyclines in mammalian cells. Science (1995) 268:1766–1769.
   PubMed Web of Science ®Google Scholar
• LIANG X, HARTIKKA J, SUKHU L, MANTHORPE M, HOBART P: Novel, high expressing and antibiotic-controlled plasmid vectors designed for use in gene therapy. Gene Ther. (1996) 3:350–356.
   PubMedGoogle Scholar
• BRACKENRIDGE S, ASHE HL, GIACCA M, PROUDFOOT NJ: Transcription and polyadenylation in a short human intergenic region. Nuclek Acids Res. (1997) 25:2326–2336.
   PubMedGoogle Scholar
• BEDWELL DM, KAENJAK A, BENOS DJ et al.: Suppression of a CEIR premature stop mutation in a bronchial epithelial cell line. Nature Med. (1997) 3:1280–1284.
   PubMed Web of Science ®Google Scholar
• KOPITO RR: Biosynthesis and degradation of CE'llt. Physic)]. Rev. (1999) 79:S167–173.
   PubMed Web of Science ®Google Scholar
• •A good introduction to the complex factors that mediate and regulate CFTR maturation and turnover.
   Google Scholar
• ZEITLIN PL: Pharmacologic restoration of AF508 CEIR-mediated chloride current. Kidney Int. (2000) 57:832–837.
   PubMed Web of Science ®Google Scholar
• CHENG SH, FANG SL, ZABNER J et al: Functional activa-tion of the cystic fibrosis trafficking mutant AF508-CEIR by overexpression. Am. J. Physiol. (1995) 2681615–24.
   Google Scholar
• NADLER SG, TEPPER MA, SCHACTER B, MAZZUCCO CE: Interaction of the immunosuppressant deoxysper-g-ualin with a member of the Hsp70 family of heat shock proteins. Science (1992) 258:484–486.
   PubMed Web of Science ®Google Scholar
• JIANG C, FANG SL, XIAO YF et al.: Partial restoration of cAMP-stimulated CEIR chloride channel activity in AF508 cells by deoxysperg-ualin. Am. J Physic)]. (1998) 275:C171–178.
   PubMedGoogle Scholar
• STEPKOWSKI SM: Molecular targets for existing andnovel immunosuppressive drugs. Exp. Rev. Molec. Med. (21 June 2000)://www-ermm.cbcu.cam.ac.uk/00001769a.pdf.
   Google Scholar
• KARACHUNSKI PI, OSTLIE NS, OKITA DK, GARMAN R, CONTI-FINE BM: Subcutaneous administration of T-epitope sequences of the acetylcholine receptor prevents experimental myasthenia gravis. J. Neuroim-munol. (1999) 93:108–121.
   PubMed Web of Science ®Google Scholar
• VARLEY AW, MUNFORD RS: Physiologically responsive gene therapy. Mol Med. Today. (1998) 4:445–451.
   PubMedGoogle Scholar
• GRIESENBACH U, SCHEID P, HILLERY E et al.: Anti-inflammatory gene therapy directed at the airway epithelium. Gene Ther. (2000) 7:306–313.
   PubMed Web of Science ®Google Scholar
• STERN M, CAPLEN NJ, BROWNING JE et al: The effect of mucolytic agents on gene transfer across a CF sputum barrier in vitro. Gene Ther. (1998) 5:91–98.
   PubMed Web of Science ®Google Scholar
• LAGASSE E, WEISSMAN IL: bc1-2 inhibits apoptosis of neutrophils but not their engulfment by macrophages. J. Exp. Med. (1994) 179:1047–1052.
   PubMed Web of Science ®Google Scholar
• ROSS GF, BRUNO MD, UYEDA M et al.: Enhanced reporter gene expression in cells transfected in the presence of DMI-2, an acid nuclease inhibitor. Gene Ther. (1998) 5:1244–1250.
   PubMed Web of Science ®Google Scholar
• CHU Q, TOUSIGNANT JD, FANG S et al: Binding and uptake of cationic lipid:pDNA complexes by polarized airway epithelial cells. Hum. Gene Ther. (1999) 10:25–36.
   PubMed Web of Science ®Google Scholar
• SMITH JJ, TRAVIS SM, GREENBERG EP, WELSH MJ: Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell (1996) 85:229–236.
   PubMed Web of Science ®Google Scholar
• ••Documents the salt-sensitivity of I3-defensins.
   Google Scholar
• ZABNER J, SMITH JJ, KARP PH, WIDDICOMBE JH, WELSH: Loss of CEIR chloride channels alters salt absorp-tion by cystic fibrosis airway epithelia in vitro. Mol Cell. (1998) 2:397–403.
   PubMed Web of Science ®Google Scholar
• MATSUI H, GRUBB BR, TARRAN R et al.: Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell (1998) 95:1005–1015.
   PubMed Web of Science ®Google Scholar
• DORIN JR, FARLEY R, WEBB S et al.: A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther. (1996) 3:797–801.
   PubMed Web of Science ®Google Scholar
• ••Key work that correlates CF phenotype with level of CF7'Rexpression in the mouse. Work such as this strengthens the idea that correction to 5-10% normal CFTR levels should be therapeutic.
   Google Scholar
• YONEMITSU Y, KITSON C, FERRARI S et al.: Efficient gene to airway epithelium using recombinant Sendai virus. Nat. Biotechnol (2000) 18:970–973.
   PubMed Web of Science ®Google Scholar
• ••Pioneering work demonstrating the potential of Sendai virusas a vehicle for CFGT.
   Google Scholar
• KRIEG AM: The role of CpG motifs in innate immunity.. Opin. Immunol. (2000) 12:35–43.
   PubMed Web of Science ®Google Scholar
• •Reviews the influence of DNA methylation status on innate immunity.
   Google Scholar
• YEW NS, WANG KX, PRZYBYLSKA M et al: Contribution plasmid DNA to inflammation in the lung after of cationic lipid:pDNA complexes. Hum. Gene Ther. (1999) 10:223–234.
   PubMed Web of Science ®Google Scholar
• BOYD AC, DAVIDSON HE, DOHERTY A et al: Construc-tion and characterisation of genomic context vectors for CF gene therapy (POS11R ABSTRACT). Ped. Pulm. (1999) S19:237.
   Google Scholar
• YE S, COLE-STRAUSS AC, FRANK B, KMIEC EB: Targeted gene correction: a new strategy for molecular medicine. Mol Med. Today (1998) 4:431–437.
   PubMedGoogle Scholar
• •Review of a novel method using short DNA/RNA oligonu-cleotides to correct mutations.
   Google Scholar
• ZEITLIN PL: Novel pharmacologic therapies for cystic fibrosis. J. Clin. Invest. (1999) 103 :447–452.
   PubMed Web of Science ®Google Scholar
• Gene therapy: cautious optimism (Editorial). Nature Med. (2000) 6:717.
   PubMedGoogle Scholar
• •Editorial signalling recent clinical successes of GT.
   Google Scholar