Treatment of pulmonary fibrosis with siRNA against a collagen-specific chaperone HSP47 in vitamin A-coupled liposomes

References

• Raghu G, Collard HR, Egan JJ, Martinez FJ, Behr J, Brown KK, et al. An official ATS/ERS/JRS/ALAT statement. Idiopathic pulmonary fibrosis: evidence-based guidelines for diagnosis and management. Am J Respir Crit Care Med. 2011;183:788–824.
   PubMed Web of Science ®Google Scholar
• Natsuizaka M, Chiba H, Kuronuma K, Otsuka M, Kudo K, Mori M, et al. Epidemiologic survey of Japanese patients with idiopathic pulmonary fibrosis and investigation of ethnic differences. Am J Respir Crit Care Med. 2014;190:773–779.
   PubMed Web of Science ®Google Scholar
• Selman M, King TE, Pardo A. Idiopathic pulmonary fibrosis: prevailing and evolving hypotheses about its pathogenesis and implications for therapy. Ann Intern Med. 2001;134:136–151.
   PubMed Web of Science ®Google Scholar
• Noble PW, Albera C, Bradford WZ, Costabel U, du Bois RM, Fagan EA, et al. Pirfenidone for idiopathic pulmonary fibrosis: analysis of pooled data from three multinational phase 3 trials. Eur Respir J. 2016;47:243–253.
   PubMed Web of Science ®Google Scholar
• Richeldi L, Cottin V, du Bois RM, Selman M, Kimura T, Bailes Z, et al. Nintedanib in patients with idiopathic pulmonary fibrosis: combined evidence from the TOMORROW and INPULSIS® trials. Respir Med. 2016;113:74–79.
   PubMed Web of Science ®Google Scholar
• Collard HR, King TE Jr. Demystifying idiopathic interstitial pneumonia. Arch Intern Med. 2003;163:17–29.
   PubMedGoogle Scholar
• Dafforn TR, Della M, Miller AD. The molecular interactions of heat shock protein 47 (Hsp47) and their implications for collagen biosynthesis. J Biol Chem. 2001;276:49310–49319.
   PubMed Web of Science ®Google Scholar
• Nakai A, Satoh M, Hirayoshi K, Nagata K. Involvement of the stress protein HSP47 in procollagen processing in the endoplasmic reticulum. J Cell Biol. 1992;117:903–914.
   PubMed Web of Science ®Google Scholar
• Nagata K. HSP47 as a collagen-specific molecular chaperone: function and expression in normal mouse development. Semin Cell Dev Biol. 2003;14:275–282.
   PubMed Web of Science ®Google Scholar
• Sato Y, Murase K, Kato J, Kobune M, Sato T, Kawano Y, et al. Resolution of liver cirrhosis using vitamin A-coupled liposomes to deliver siRNA against a collagen-specific chaperone. Nat Biotechnol. 2008;26:431–442.
   PubMed Web of Science ®Google Scholar
• Ishiwatari H, Sato Y, Murase K, Yoneda A, Fujita R, Nishita H, et al. Treatment of pancreatic fibrosis with siRNA against a collagen-specific chaperone in vitamin A-coupled liposomes. Gut. 2013;62:1328–1339.
   PubMed Web of Science ®Google Scholar
• Hagiwara S, Iwasaka H, Matsumoto S, Noguchi T. Antisense oligonucleotide inhibition of heat shock protein (HSP) 47 improves bleomycin-induced pulmonary fibrosis in rats. Respir Res 2007;8:37.
   PubMed Web of Science ®Google Scholar
• Hagiwara S, Iwasaka H, Matsumoto S, Noguchi T. Introduction of antisense oligonucleotides to heat shock protein 47 prevents pulmonary fibrosis in lipopolysaccharide-induced pneumopathy of the rat. Eur J Pharmacol. 2007;564:174–180.
   PubMed Web of Science ®Google Scholar
• Hagiwara S, Iwasaka H, Matsumoto S, Noguchi T. An antisense oligonucleotide to HSP47 inhibits paraquat-induced pulmonary fibrosis in rats. Toxicology. 2007;236:199–207.
   PubMed Web of Science ®Google Scholar
• Nishino T, Miyazaki M, Abe K, Furusu A, Mishima Y, Harada T, et al. Antisense oligonucleotides against collagen-binding stress protein HSP47 suppress peritoneal fibrosis in rats. Kidney Int. 2003;64:887–896.
   PubMed Web of Science ®Google Scholar
• Xia Z, Abe K, Furusu A, Miyazaki M, Obata Y, Tabata Y, et al. Suppression of renal tubulointerstitial fibrosis by small interfering RNA targeting heat shock protein 47. Am J Nephrol. 2008;28:34–46.
   PubMed Web of Science ®Google Scholar
• Birukawa NK, Murase K, Sato Y, Kosaka A, Yoneda A, Nishita H, et al. Activated hepatic stellate cells are dependent on self-collagen, cleaved by membrane type 1 matrix metalloproteinase for their growth. J Biol Chem. 2014;289:20209–20221.
   PubMed Web of Science ®Google Scholar
• Ishii H, Mukae H, Kakugawa T, Iwashita T, Kaida H, Fujii T, et al. Increased expression of collagen-binding heat shock protein 47 in murine bleomycin-induced pneumopathy. Am J Physiol Lung Cell Mol Physiol. 2003;285:L957–963.
   PubMed Web of Science ®Google Scholar
• Kakugawa T, Mukae H, Hishikawa Y, Ishii H, Sakamoto N, Ishimatsu Y, et al. Localization of HSP47 mRNA in murine bleomycin-induced pulmonary fibrosis. Virchows Arch. 2010;456:309–315.
   PubMed Web of Science ®Google Scholar
• Kakugawa T, Mukae H, Hayashi T, Ishii H, Nakayama S, Sakamoto N, et al. Expression of HSP47 in usual interstitial pneumonia and nonspecific interstitial pneumonia. Respir Res. 2005;6:57.
   PubMed Web of Science ®Google Scholar
• Razzaque MS, Nazneen A, Taguchi T. Immunolocalization of collagen and collagen-binding heat shock protein 47 in fibrotic lung diseases. Mod Pathol. 1998;11:1183–1188.
   PubMed Web of Science ®Google Scholar
• Iwashita T, Kadota J, Naito S, Kaida H, Ishimatsu Y, Miyazaki M, et al. Involvement of collagen-binding heat shock protein 47 and procollagen type I synthesis in idiopathic pulmonary fibrosis: contribution of type II pneumocytes to fibrosis. Hum Pathol. 2000;31:1498–1505.
   PubMed Web of Science ®Google Scholar
• Scotton CJ, Chambers RC. Molecular targets in pulmonary fibrosis: the myofibroblast in focus. Chest. 2007;132:1311–1321.
   PubMed Web of Science ®Google Scholar
• Darby IA, Zakuan N, Billet F, Desmoulière A. The myofibroblast, a key cell in normal and pathological tissue repair. Cell Mol Life Sci. 2016;73:1145–1157.
   PubMed Web of Science ®Google Scholar
• Friedman SL. Molecular regulation of hepatic fibrosis, an integrated cellular response to tissue injury. J Biol Chem. 2000;275:2247–2250.
   PubMed Web of Science ®Google Scholar
• Nagy NE, Holven KB, Roos N, Senoo H, Kojima N, Norum KR, et al. Storage of vitamin A in extrahepatic stellate cells in normal rats. J Lipid Res. 1997;38:645–658.
   PubMed Web of Science ®Google Scholar
• Zhao L, Burt AD. The diffuse stellate cell system. J Mol Histol. 2007;38:53–64.
   PubMed Web of Science ®Google Scholar
• Tahedl D, Wirkes A, Tschanz SA, Ochs M, Mühlfeld C. How common is the lipid body-containing interstitial cell in the mammalian lung? Am J Physiol Lung Cell Mol Physiol. 2014;307:L386–394.
   PubMed Web of Science ®Google Scholar
• Otsuka M, Takahashi H, Shiratori M, Chiba H, Abe S. Reduction of bleomycin induced lung fibrosis by candesartan cilexetil, an angiotensin II type 1 receptor antagonist. Thorax. 2004;59:31–38.
   PubMed Web of Science ®Google Scholar
• Kivirikko KI, Laitinen O, Prockop DJ. Modifications of a specific assay for hydroxyproline in urine. Anal Biochem. 1967;19:249–255.
   PubMed Web of Science ®Google Scholar
• Hubner RH, Gitter W, E Mokhtari NE, Mathiak M, Both M, Bolte H, et al. Standardized quantification of pulmonary fibrosis in histological samples. BioTechniques. 2008;44:507–511, 514–517.
   PubMed Web of Science ®Google Scholar
• Judge AD, Sood V, Shaw JR, Fang D, McClintock K, MacLachlan I. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nat Biotechnol. 2005;23:457–462.
   PubMed Web of Science ®Google Scholar
• Jackson AL, Bartz SR, Schelter J, Kobayashi SV, Burchard J, Mao M, et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol. 2003;21:635–637.
   PubMed Web of Science ®Google Scholar
• Ibaraki H, Kanazawa T, Takashima Y, Okada H, Seta Y. Development of an innovative intradermal siRNA delivery system using a combination of a functional stearylated cytoplasm-responsive peptide and a tight junction-opening peptide. Molecules. 2016;21:1279.
   PubMed Web of Science ®Google Scholar
• Park AM, Kanai K, Itoh T, Sato T, Tsukui T, Inagaki Y, et al. Heat shock protein 27 plays a pivotal role in myofibroblast differentiation and in the development of bleomycin-induced pulmonary fibrosis. PLoS One. 2016;11:e0148998.
   PubMed Web of Science ®Google Scholar
• Sontake V, Wang Y, Kasam RK, Sinner D, Reddy GB, Naren AP, et al. Hsp90 regulation of fibroblast activation in pulmonary fibrosis. J CI Insight. 2017;2:e91454.
   Google Scholar
• Kawasaki K, Ushioda R, Ito S, Ikeda K, Masago Y, Nagata K. Deletion of the collagen-specific molecular chaperone Hsp47 causes endoplasmic reticulum stress-mediated apoptosis of hepatic stellate cells. J Biol Chem. 2015;290:3639–3646.
   PubMed Web of Science ®Google Scholar
• Ramos C, Montaño M, García-Alvarez J, Ruiz V, Uhal BD, Selman M, et al. Fibroblasts from idiopathic pulmonary fibrosis and normal lungs differ in growth rate, apoptosis, and tissue inhibitor of metalloproteinases expression. Am J Respir Cell Mol Biol. 2001;24:591–598.
   PubMed Web of Science ®Google Scholar
• Selman M, Ruiz V, Cabrera S, Segura L, Ramírez R, Barrios R, et al. TIMP-1, −2, −3, and −4 in idiopathic pulmonary fibrosis. A prevailing nondegradative lung microenvironment? Am J Physiol Lung Cell Mol Physiol. 2000;279:L562–574.
   PubMed Web of Science ®Google Scholar
• Kendall RT, Feghali-Bostwick CA. Fibroblasts in fibrosis: novel roles and mediators. Front Pharmacol. 2014;5:123.
   PubMed Web of Science ®Google Scholar
• Gharaee-Kermani M, Phan SH. Role of Fibroblasts and Myofibroblasts in Idiopathic Pulmonary Fibrosis. In: Lynch JP, ed. 3rd editors. Idiopathic Pulmonary Fibrosis. Vol. 185. New York: Marcel Dekker; 2004.
   Google Scholar
• Dirami G, Massaro GD, Clerch LB, Ryan US, Reczek PR, Massaro D. Lung retinol storing cells synthesize and secrete retinoic acid, an inducer of alveolus formation. Am J Physiol Lung Cell Mol Physiol. 2004;286:L249–L256.
   PubMed Web of Science ®Google Scholar
• Maksvytis HJ, Niles RM, Simanovsky L, Minassian IA, Richardson LL, Hamosh M, et al. In vitro characteristics of the lipid-filled interstitial cell associated with postnatal lung growth: evidence for fibroblast heterogeneity. J Cell Physiol. 1984;118:113–123.
   PubMed Web of Science ®Google Scholar