Utilisation de l’ARN interférence dans le traitement de l’amylose héréditaire due à une mutation de la transthyrétine

David Adams; Pierre Lozeron; Vincent Algalarrondo

doi:10.1051/medsci/20143004002

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Volume 30 / Numéro 4 (Avril 2014)

Med Sci (Paris), 30 4 (2014) 345-347

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Numéro		Med Sci (Paris) Volume 30, Numéro 4, Avril 2014


Page(s)		345 - 347
Section		Nouvelles
DOI		https://doi.org/10.1051/medsci/20143004002
Publié en ligne		5 mai 2014

Adams D, Lozeron P, Theaudin M, et al. Regional difference and similarity of familial amyloidosis with polyneuropathy in France. Amyloid 2012 ; 19 Suppl 1 : 61–64. [CrossRef] [Google Scholar]
Adams D, Samuel D, Goulon-Goeau C, et al. The course, prognostic factors of familial amyloid polyneuropathy after liver transplantation. Brain 2000 ; 123 (Pt 7) : 1495–1504. [CrossRef] [PubMed] [Google Scholar]
Wilczek HE, Larsson M, Ericzon BG, Fapwtr. Long-term data from the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR). Amyloid 2011 ; 18 Suppl 1 : 193–195. [CrossRef] [PubMed] [Google Scholar]
Yamashita T, Ando Y, Okamoto S, et al. Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology 2012 ; 78 : 637–643. [CrossRef] [PubMed] [Google Scholar]
Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology 2010 ; 75 : 324–327. [CrossRef] [PubMed] [Google Scholar]
Yazaki M, Mitsuhashi S, Tokuda T, et al. Progressive wild-type transthyretin deposition after liver transplantation preferentially occurs onto myocardium in FAP patients. Am J Transplant 2007 ; 7 : 235–242. [CrossRef] [PubMed] [Google Scholar]
Ihse E, Suhr OB, Hellman U, Westermark P. Variation in amount of wild-type transthyretin in different fibril and tissue types in ATTR amyloidosis. J Mol Med 2011 ; 89 : 171–180. [CrossRef] [PubMed] [Google Scholar]
Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy a randomized, controlled trial. Neurology 2012 ; 79 : 785–792. [CrossRef] [PubMed] [Google Scholar]
Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001 ; 411 : 494–498. [CrossRef] [PubMed] [Google Scholar]
Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. New Engl J Med 2013 ; 369 : 819–829. [CrossRef] [Google Scholar]

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[1] Adams D, Lozeron P, Theaudin M, et al. Regional difference and similarity of familial amyloidosis with polyneuropathy in France. Amyloid 2012 ; 19 Suppl 1 : 61–64. [CrossRef] [Google Scholar]

[2] Adams D, Samuel D, Goulon-Goeau C, et al. The course, prognostic factors of familial amyloid polyneuropathy after liver transplantation. Brain 2000 ; 123 (Pt 7) : 1495–1504. [CrossRef] [PubMed] [Google Scholar]

[3] Wilczek HE, Larsson M, Ericzon BG, Fapwtr. Long-term data from the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR). Amyloid 2011 ; 18 Suppl 1 : 193–195. [CrossRef] [PubMed] [Google Scholar]

[4] Yamashita T, Ando Y, Okamoto S, et al. Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology 2012 ; 78 : 637–643. [CrossRef] [PubMed] [Google Scholar]

[5] Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology 2010 ; 75 : 324–327. [CrossRef] [PubMed] [Google Scholar]

[6] Yazaki M, Mitsuhashi S, Tokuda T, et al. Progressive wild-type transthyretin deposition after liver transplantation preferentially occurs onto myocardium in FAP patients. Am J Transplant 2007 ; 7 : 235–242. [CrossRef] [PubMed] [Google Scholar]

[7] Ihse E, Suhr OB, Hellman U, Westermark P. Variation in amount of wild-type transthyretin in different fibril and tissue types in ATTR amyloidosis. J Mol Med 2011 ; 89 : 171–180. [CrossRef] [PubMed] [Google Scholar]

[8] Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy a randomized, controlled trial. Neurology 2012 ; 79 : 785–792. [CrossRef] [PubMed] [Google Scholar]

[9] Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001 ; 411 : 494–498. [CrossRef] [PubMed] [Google Scholar]

[10] Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. New Engl J Med 2013 ; 369 : 819–829. [CrossRef] [Google Scholar]