Número do ensaio clínico NCT01960348 para "APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis" en ClinicalTrials.gov
Razavi H, Palaninathan SK, Powers ET, Wiseman RL, Purkey HE, Mohamedmohaideen NN, Deechongkit S, Chiang KP, Dendle MT, Sacchettini JC, Kelly JW (xuño de 2003). "Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action". Angew. Chem. Int. Ed. Engl.42 (24): 2758–61. PMID12820260. doi:10.1002/anie.200351179.
Sekijima Y, Dendle MA, Kelly JW (decembro de 2006). "Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis". Amyloid13 (4): 236–49. PMID17107884. doi:10.1080/13506120600960882.
Adamski-Werner SL, Palaninathan SK, Sacchettini JC, Kelly JW (xaneiro de 2004). "Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis". J. Med. Chem.47 (2): 355–74. PMID14711308. doi:10.1021/jm030347n.
Vilaro M, Arsequell G, Valencia G, Ballesteros A, Barluenga J, Nieto J, Planas A, Almeida R, Saraiva MJ (2007). "Reengineering TTR amyloid inhibition properties of diflunisal". En Seldin DC, Skinner M, Berk JL, Connors LH. XIth International Symposium on Amyloidosis. Boca Raton: CRC. pp. 205–207. ISBN978-1-4200-4281-8. doi:10.1201/9781420043358.ch69.
Baures PW, Oza VB, Peterson SA, Kelly JW (xullo de 1999). "Synthesis and evaluation of inhibitors of transthyretin amyloid formation based on the non-steroidal anti-inflammatory drug, flufenamic acid". Bioorg. Med. Chem.7 (7): 1339–47. PMID10465408. doi:10.1016/S0968-0896(99)00066-8.
Purkey HE, Palaninathan SK, Kent KC, Smith C, Safe SH, Sacchettini JC, Kelly JW (decembro de 2004). "Hydroxylated polychlorinated biphenyls selectively bind transthyretin in blood and inhibit amyloidogenesis: rationalizing rodent PCB toxicity". Chem. Biol.11 (12): 1719–28. PMID15610856. doi:10.1016/j.chembiol.2004.10.009.
Foss TR, Wiseman RL, Kelly JW (novembor de 2005). "The pathway by which the tetrameric protein transthyretin dissociates". Biochemistry44 (47): 15525–33. PMID16300401. doi:10.1021/bi051608t.
Andrade C (setembro de 1952). "A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves". Brain75 (3): 408–27. PMID12978172. doi:10.1093/brain/75.3.408.
Coelho T (outubro de 1996). "Familial amyloid polyneuropathy: new developments in genetics and treatment". Curr. Opin. Neurol.9 (5): 355–9. PMID8894411. doi:10.1097/00019052-199610000-00007.
Jacobson DR, Pastore RD, Yaghoubian R, Kane I, Gallo G, Buck FS, Buxbaum JN (febreiro de 1997). "Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans". N. Engl. J. Med.336 (7): 466–73. PMID9017939. doi:10.1056/NEJM199702133360703.
Colon W, Kelly JW (setembro de 1992). "Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro". Biochemistry31 (36): 8654–60. PMID1390650. doi:10.1021/bi00151a036.
Lai Z, Colón W, Kelly JW (maio de 1996). "The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid". Biochemistry35 (20): 6470–82. PMID8639594. doi:10.1021/bi952501g.
Jiang X, Smith CS, Petrassi HM, Hammarström P, White JT, Sacchettini JC, Kelly JW (setembro de 2001). "An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured". Biochemistry40 (38): 11442–52. PMID11560492. doi:10.1021/bi011194d.
Sekijima Y, Wiseman RL, Matteson J, Hammarström P, Miller SR, Sawkar AR, Balch WE, Kelly JW (abril de 2005). "The biological and chemical basis for tissue-selective amyloid disease". Cell121 (1): 73–85. PMID15820680. doi:10.1016/j.cell.2005.01.018.
Holmgren G, Ericzon BG, Groth CG, Steen L, Suhr O, Andersen O, Wallin BG, Seymour A, Richardson S, Hawkins PN (maio de 1993). "Clinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosis". Lancet341 (8853): 1113–6. PMID8097803. doi:10.1016/0140-6736(93)93127-m.
Ando Y, Suhr OB (decembro de 1998). "Autonomic dysfunction in familial amyloidotic polyneuropathy (FAP)". Amyloid5 (4): 288–300. PMID10036588. doi:10.3109/13506129809007303.
Razavi H, Palaninathan SK, Powers ET, Wiseman RL, Purkey HE, Mohamedmohaideen NN, Deechongkit S, Chiang KP, Dendle MT, Sacchettini JC, Kelly JW (xuño de 2003). "Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action". Angew. Chem. Int. Ed. Engl.42 (24): 2758–61. PMID12820260. doi:10.1002/anie.200351179.
Sekijima Y, Dendle MA, Kelly JW (decembro de 2006). "Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis". Amyloid13 (4): 236–49. PMID17107884. doi:10.1080/13506120600960882.
Adamski-Werner SL, Palaninathan SK, Sacchettini JC, Kelly JW (xaneiro de 2004). "Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis". J. Med. Chem.47 (2): 355–74. PMID14711308. doi:10.1021/jm030347n.
Baures PW, Oza VB, Peterson SA, Kelly JW (xullo de 1999). "Synthesis and evaluation of inhibitors of transthyretin amyloid formation based on the non-steroidal anti-inflammatory drug, flufenamic acid". Bioorg. Med. Chem.7 (7): 1339–47. PMID10465408. doi:10.1016/S0968-0896(99)00066-8.
Purkey HE, Palaninathan SK, Kent KC, Smith C, Safe SH, Sacchettini JC, Kelly JW (decembro de 2004). "Hydroxylated polychlorinated biphenyls selectively bind transthyretin in blood and inhibit amyloidogenesis: rationalizing rodent PCB toxicity". Chem. Biol.11 (12): 1719–28. PMID15610856. doi:10.1016/j.chembiol.2004.10.009.
Foss TR, Wiseman RL, Kelly JW (novembor de 2005). "The pathway by which the tetrameric protein transthyretin dissociates". Biochemistry44 (47): 15525–33. PMID16300401. doi:10.1021/bi051608t.
Andrade C (setembro de 1952). "A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves". Brain75 (3): 408–27. PMID12978172. doi:10.1093/brain/75.3.408.
Coelho T (outubro de 1996). "Familial amyloid polyneuropathy: new developments in genetics and treatment". Curr. Opin. Neurol.9 (5): 355–9. PMID8894411. doi:10.1097/00019052-199610000-00007.
Jacobson DR, Pastore RD, Yaghoubian R, Kane I, Gallo G, Buck FS, Buxbaum JN (febreiro de 1997). "Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans". N. Engl. J. Med.336 (7): 466–73. PMID9017939. doi:10.1056/NEJM199702133360703.
Colon W, Kelly JW (setembro de 1992). "Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro". Biochemistry31 (36): 8654–60. PMID1390650. doi:10.1021/bi00151a036.
Lai Z, Colón W, Kelly JW (maio de 1996). "The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid". Biochemistry35 (20): 6470–82. PMID8639594. doi:10.1021/bi952501g.
Jiang X, Smith CS, Petrassi HM, Hammarström P, White JT, Sacchettini JC, Kelly JW (setembro de 2001). "An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured". Biochemistry40 (38): 11442–52. PMID11560492. doi:10.1021/bi011194d.
Sekijima Y, Wiseman RL, Matteson J, Hammarström P, Miller SR, Sawkar AR, Balch WE, Kelly JW (abril de 2005). "The biological and chemical basis for tissue-selective amyloid disease". Cell121 (1): 73–85. PMID15820680. doi:10.1016/j.cell.2005.01.018.
Holmgren G, Ericzon BG, Groth CG, Steen L, Suhr O, Andersen O, Wallin BG, Seymour A, Richardson S, Hawkins PN (maio de 1993). "Clinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosis". Lancet341 (8853): 1113–6. PMID8097803. doi:10.1016/0140-6736(93)93127-m.
Ando Y, Suhr OB (decembro de 1998). "Autonomic dysfunction in familial amyloidotic polyneuropathy (FAP)". Amyloid5 (4): 288–300. PMID10036588. doi:10.3109/13506129809007303.
Albrecht, S., Rouah, E., Becker, L. E., & Bruner, J. (1991). Transthyretin immunoreactivity in choroid plexus neoplasms and brain metastases. Modern pathology : an official journal of the United States and Canadian Academy of Pathology, Inc, 4(5), 610–614. PMID 1758873
