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Recent advances in transthyretin amyloidosis therapy. Transl. Neurodegener.<\/i>, 3<\/b>, 19 (2014).","DOI":"10.1186\/2047-9158-3-19"},{"key":"2","doi-asserted-by":"crossref","unstructured":"2) Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch. Neurol.<\/i>, 62<\/b>, 1057\u20131062 (2005).","DOI":"10.1001\/archneur.62.7.1057"},{"key":"3","doi-asserted-by":"crossref","unstructured":"3) Gasperini RJ, Klaver DW, Hou X, Aguilar MI, Small DH. Mechanisms of transthyretin aggregation and toxicity. Subcell. Biochem.<\/i>, 65<\/b>, 211\u2013224 (2012).","DOI":"10.1007\/978-94-007-5416-4_9"},{"key":"4","doi-asserted-by":"crossref","unstructured":"4) Saelices L, Johnson LM, Liang WY, Sawaya MR, Cascio D, Ruchala P, Whitelegge J, Jiang L, Riek R, Eisenberg DS. Uncovering the mechanism of aggregation of human transthyretin. J. Biol. Chem.<\/i>, 290<\/b>, 28932\u201328943 (2015).","DOI":"10.1074\/jbc.M115.659912"},{"key":"5","doi-asserted-by":"crossref","unstructured":"5) Hara R, Kawaji T, Ando E, Ohya Y, Ando Y, Tanihara H. Impact of liver transplantation on transthyretin-related ocular amyloidosis in Japanese patients. Arch. Ophthalmol.<\/i>, 128<\/b>, 206\u2013210 (2010).","DOI":"10.1001\/archophthalmol.2009.390"},{"key":"6","doi-asserted-by":"crossref","unstructured":"6) Koga T, Ando E, Hirata A, Fukushima M, Kimura A, Ando Y, Negi A, Tanihara H. Vitreous opacities and outcome of vitreous surgery in patients with familial amyloidotic polyneuropathy. Am. J. Ophthalmol.<\/i>, 135<\/b>, 188\u2013193 (2003).","DOI":"10.1016\/S0002-9394(02)01838-X"},{"key":"7","doi-asserted-by":"crossref","unstructured":"7) Kimura A, Ando E, Fukushima M, Koga T, Hirata A, Arimura K, Ando Y, Negi A, Tanihara H. Secondary glaucoma in patients with familial amyloidotic polyneuropathy. Arch. Ophthalmol.<\/i>, 121<\/b>, 351\u2013356 (2003).","DOI":"10.1001\/archopht.121.3.351"},{"key":"8","doi-asserted-by":"crossref","unstructured":"8) Kawaji T, Ando Y, Nakamura M, Yamamoto K, Ando E, Takano A, Inomata Y, Hirata A, Tanihara H. Transthyretin synthesis in rabbit ciliary pigment epithelium. Exp. Eye Res.<\/i>, 81<\/b>, 306\u2013312 (2005).","DOI":"10.1016\/j.exer.2005.02.003"},{"key":"9","doi-asserted-by":"crossref","unstructured":"9) Kawaji T, Inoue T, Hara R, Eiki D, Ando Y, Tanihara H. Long-term outcomes and complications of trabeculectomy for secondary glaucoma in patients with familial amyloidotic polyneuropathy. PLOS ONE<\/i>, 9<\/b>, e96324 (2014).","DOI":"10.1371\/journal.pone.0096324"},{"key":"10","doi-asserted-by":"crossref","unstructured":"10) Kawaji T, Ando Y, Hara R, Tanihara H. Novel therapy for transthyretin-related ocular amyloidosis: a pilot study of retinal laser photocoagulation. Ophthalmology<\/i>, 117<\/b>, 552\u2013555 (2010).","DOI":"10.1016\/j.ophtha.2009.07.042"},{"key":"11","doi-asserted-by":"crossref","unstructured":"11) Yang X. Applications of CRISPR-Cas9 mediated genome engineering. Mil. Med. Res.<\/i>, 2<\/b>, 11 (2015).","DOI":"10.1186\/s40779-015-0038-1"},{"key":"12","doi-asserted-by":"crossref","unstructured":"12) Savi\u0107 N, Schwank G. Advances in therapeutic CRISPR\/Cas9 genome editing. Transl. Res.<\/i>, 168<\/b>, 15\u201321 (2016).","DOI":"10.1016\/j.trsl.2015.09.008"},{"key":"13","doi-asserted-by":"crossref","unstructured":"13) Seah YF, El Farran CA, Warrier T, Xu J, Loh YH. Induced pluripotency and gene editing in disease modelling: perspectives and challenges. Int. J. Mol. Sci.<\/i>, 16<\/b>, 28614\u201328634 (2015).","DOI":"10.3390\/ijms161226119"},{"key":"14","doi-asserted-by":"crossref","unstructured":"14) Nishimasu H, Ran FA, Hsu PD, Konermann S, Shehata SI, Dohmae N, Ishitani R, Zhang F, Nureki O. Crystal structure of Cas9 in complex with guide RNA and target DNA. Cell<\/i>, 156<\/b>, 935\u2013949 (2014).","DOI":"10.1016\/j.cell.2014.02.001"},{"key":"15","doi-asserted-by":"crossref","unstructured":"15) Liu C, Zhang L, Liu H, Cheng K. Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications. J. Control. Release<\/i>, 266<\/b>, 17\u201326 (2017).","DOI":"10.1016\/j.jconrel.2017.09.012"},{"key":"16","doi-asserted-by":"crossref","unstructured":"16) Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, Norville JE, Church GM. RNA-guided human genome engineering via<\/i> Cas9. Science<\/i>, 339<\/b>, 823\u2013826 (2013).","DOI":"10.1126\/science.1232033"},{"key":"17","doi-asserted-by":"crossref","unstructured":"17) Shalem O, Sanjana NE, Hartenian E, Shi X, Scott DA, Mikkelson T, Heckl D, Ebert BL, Root DE, Doench JG, Zhang F. Genome-scale CRISPR-Cas9 knockout screening in human cells. Science<\/i>, 343<\/b>, 84\u201387 (2014).","DOI":"10.1126\/science.1247005"},{"key":"18","doi-asserted-by":"crossref","unstructured":"18) Bridges CC, El-Sherbeny A, Ola MS, Ganapathy V, Smith SB. Transcellular transfer of folate across the retinal pigment epithelium. Curr. Eye Res.<\/i>, 24<\/b>, 129\u2013138 (2002).","DOI":"10.1076\/ceyr.24.2.129.8167"},{"key":"19","doi-asserted-by":"crossref","unstructured":"19) Antony AC. The biological chemistry of folate receptors. Blood<\/i>, 79<\/b>, 2807\u20132820 (1992).","DOI":"10.1182\/blood.V79.11.2807.2807"},{"key":"20","doi-asserted-by":"crossref","unstructured":"20) Kelemen LE. The role of folate receptor alpha in cancer development, progression and treatment: cause, consequence or innocent bystander? Int. J. Cancer<\/i>, 119<\/b>, 243\u2013250 (2006).","DOI":"10.1002\/ijc.21712"},{"key":"21","doi-asserted-by":"crossref","unstructured":"21) Salazar MD, Ratnam M. The folate receptor: what does it promise in tissue-targeted therapeutics? Cancer Metastasis Rev.<\/i>, 26<\/b>, 141\u2013152 (2007).","DOI":"10.1007\/s10555-007-9048-0"},{"key":"22","doi-asserted-by":"crossref","unstructured":"22) Anno T, Higashi T, Motoyama K, Hirayama F, Uekama K, Arima H. Potential use of glucuronylglucosyl-\u03b2-cyclodextrin\/dendrimer conjugate (G2) as a DNA carrier in vitro<\/i> and in vivo.<\/i> J. Drug Target.<\/i>, 20<\/b>, 272\u2013280 (2012).","DOI":"10.3109\/1061186X.2011.645163"},{"key":"23","doi-asserted-by":"crossref","unstructured":"23) Inoue M, Ueda M, Higashi T, Anno T, Fujisawa K, Motoyama K, Mizuguchi M, Ando Y, Jono H, Arima H. Therapeutic potential of polyamidoamine dendrimer for amyloidogenic transthyretin amyloidosis. ACS Chem. Neurosci.<\/i>, 10<\/b>, 2584\u20132590 (2019).","DOI":"10.1021\/acschemneuro.9b00059"},{"key":"24","doi-asserted-by":"crossref","unstructured":"24) Kim SS, Garg H, Joshi A, Manjunath N. Strategies for targeted nonviral delivery of siRNAs in vivo.<\/i> Trends Mol. Med.<\/i>, 15<\/b>, 491\u2013500 (2009).","DOI":"10.1016\/j.molmed.2009.09.001"},{"key":"25","doi-asserted-by":"crossref","unstructured":"25) Ohyama A, Higashi T, Motoyama K, Arima H. In vitro<\/i> and in vivo<\/i> tumor-targeting siRNA delivery using folate-PEG-appended dendrimer (G4)\/\u03b1-cyclodextrin conjugates. Bioconjug. Chem.<\/i>, 27<\/b>, 521\u2013532 (2016).","DOI":"10.1021\/acs.bioconjchem.5b00545"},{"key":"26","doi-asserted-by":"crossref","unstructured":"26) Aramaki Y, Takano S, Tsuchiya S. Induction of apoptosis in macrophages by cationic liposomes. FEBS Lett.<\/i>, 460<\/b>, 472\u2013476 (1999).","DOI":"10.1016\/S0014-5793(99)01386-1"},{"key":"27","doi-asserted-by":"crossref","unstructured":"27) Fischer D, Li Y, Ahlemeyer B, Krieglstein J, Kissel T. In vitro<\/i> cytotoxicity testing of polycations: influence of polymer structure on cell viability and hemolysis. Biomaterials<\/i>, 24<\/b>, 1121\u20131131 (2003).","DOI":"10.1016\/S0142-9612(02)00445-3"},{"key":"28","doi-asserted-by":"crossref","unstructured":"28) Ibrahim M, Saint Croix GR, Lacy S, Fattouh M, Barillas-Lara MI, Behrooz L, Mechanic O. The use of diflunisal for transthyretin cardiac amyloidosis: a review. Heart Fail. Rev.<\/i>, 27<\/b>, 517\u2013524 (2022).","DOI":"10.1007\/s10741-021-10143-4"},{"key":"29","doi-asserted-by":"crossref","unstructured":"29) Cardoso I, Martins D, Ribeiro T, Merlini G, Saraiva MJ. Synergy of combined doxycycline\/TUDCA treatment in lowering Transthyretin deposition and associated biomarkers: studies in FAP mouse models. J. Transl. Med.<\/i>, 8<\/b>, 74 (2010).","DOI":"10.1186\/1479-5876-8-74"},{"key":"30","doi-asserted-by":"crossref","unstructured":"30) Abedi-Gaballu F, Dehghan G, Ghaffari M, Yekta R, Abbaspour-Ravasjani S, Baradaran B, Dolatabadi JEN, Hamblin MR. PAMAM dendrimers as efficient drug and gene delivery nanosystems for cancer therapy. Appl. Mater. Today<\/i>, 12<\/b>, 177\u2013190 (2018).","DOI":"10.1016\/j.apmt.2018.05.002"},{"key":"31","doi-asserted-by":"crossref","unstructured":"31) Reddy JA, Low PS. Folate-mediated targeting of therapeutic and imaging agents to cancers. Crit. Rev. Ther. Drug Carrier Syst.<\/i>, 15<\/b>, 587\u2013627 (1998).","DOI":"10.1615\/CritRevTherDrugCarrierSyst.v15.i6.20"},{"key":"32","doi-asserted-by":"crossref","unstructured":"32) Kihara F, Arima H, Tsutsumi T, Hirayama F, Uekama K. Effects of structure of polyamidoamine dendrimer on gene transfer efficiency of the dendrimer conjugate with alpha-cyclodextrin. Bioconjug. Chem.<\/i>, 13<\/b>, 1211\u20131219 (2002).","DOI":"10.1021\/bc025557d"},{"key":"33","doi-asserted-by":"crossref","unstructured":"33) Arima H, Chihara Y, Arizono M, Yamashita S, Wada K, Hirayama F, Uekama K. Enhancement of gene transfer activity mediated by mannosylated dendrimer\/\u03b1-cyclodextrin conjugate (generation 3, G3). J. Control. Release<\/i>, 116<\/b>, 64\u201374 (2006).","DOI":"10.1016\/j.jconrel.2006.08.026"},{"key":"34","doi-asserted-by":"crossref","unstructured":"34) Utatsu K, Kogo T, Taharabaru T, Onodera R, Motoyama K, Higashi T. Supramolecular polymer-based transformable material for reversible PEGylation of protein drugs. Mater. Today Bio.<\/i>, 12<\/b>, 100160 (2021).","DOI":"10.1016\/j.mtbio.2021.100160"},{"key":"35","unstructured":"35) Smith SB, Kekuda R, Gu X, Chancy C, Conway SJ, Ganapathy V. Expression of folate receptor alpha in the mammalian retinol pigmented epithelium and retina. Invest. Ophthalmol. Vis. Sci.<\/i>, 40<\/b>, 840\u2013848 (1999)."},{"key":"36","doi-asserted-by":"crossref","unstructured":"36) Bozard BR, Ganapathy PS, Duplantier J, Mysona B, Ha Y, Roon P, Smith R, Goldman ID, Prasad P, Martin PM, Ganapathy V, Smith SB. Molecular and biochemical characterization of folate transport proteins in retinal M\u00fcller cells. Invest. Ophthalmol. Vis. Sci.<\/i>, 51<\/b>, 3226\u20133235 (2010).","DOI":"10.1167\/iovs.09-4833"},{"key":"37","doi-asserted-by":"crossref","unstructured":"37) Luong D, Sau S, Kesharwani P, Iyer AK. Polyvalent folate-dendrimer-coated iron oxide theranostic nanoparticles for simultaneous magnetic resonance imaging and precise cancer cell targeting. 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