遺伝性トランスサイレチンアミロイドーシス治療の現状と展望
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| Published in | 神経治療学 Vol. 38; no. 3; pp. 370 - 373 |
|---|---|
| Main Author | |
| Format | Journal Article |
| Language | Japanese |
| Published |
日本神経治療学会
2021
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| Subjects | |
| Online Access | Get full text |
| ISSN | 0916-8443 2189-7824 |
| DOI | 10.15082/jsnt.38.3_370 |
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| Author | 植田, 光晴 |
|---|---|
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| Copyright | 2021 日本神経治療学会 |
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| References | 5) Adams D, Gonzalez–Duarte A, O’Riordan WD et al : Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 379 : 11–21, 2018 2) Benson MD, Buxbaum JN, Eisenberg DS et al : Amyloid nomenclature 2020 : update and recommendations by the International Society of Amyloidosis (ISA) nomenclature committee. Amyloid 27 : 217–222, 2020 6) Finn JD, Smith AR, Patel MC et al : A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing. Cell Rep 22 : 2227–2235, 2018 4) Coelho T, Maia LF, Martins da Silva A et al : Tafamidis for transthyretin familial amyloid polyneuropathy : a randomized, controlled trial. Neurology 79 : 785–792, 2012 7) Ueda M, Okada M, Mizuguchi M et al : A cell–based high–throughput screening method to directly examine transthyretin amyloid fibril formation at neutral pH. J Biol Chem 294 : 11259–11275, 2019 1) Ueda M, Ando Y : Recent advances in transthyretin amyloidosis therapy. Transl Neurodegener 3 : 19, 2014 3) Yamashita T, Ueda M, Misumi Y et al : Genetic and clinical characteristics of hereditary transthyretin amyloidosis in endemic and non–endemic areas : experience from a singlereferral center in Japan. J Neurol 265 : 134–140, 2018 |
| References_xml | – reference: 2) Benson MD, Buxbaum JN, Eisenberg DS et al : Amyloid nomenclature 2020 : update and recommendations by the International Society of Amyloidosis (ISA) nomenclature committee. Amyloid 27 : 217–222, 2020 – reference: 6) Finn JD, Smith AR, Patel MC et al : A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing. Cell Rep 22 : 2227–2235, 2018 – reference: 4) Coelho T, Maia LF, Martins da Silva A et al : Tafamidis for transthyretin familial amyloid polyneuropathy : a randomized, controlled trial. Neurology 79 : 785–792, 2012 – reference: 7) Ueda M, Okada M, Mizuguchi M et al : A cell–based high–throughput screening method to directly examine transthyretin amyloid fibril formation at neutral pH. J Biol Chem 294 : 11259–11275, 2019 – reference: 1) Ueda M, Ando Y : Recent advances in transthyretin amyloidosis therapy. Transl Neurodegener 3 : 19, 2014 – reference: 5) Adams D, Gonzalez–Duarte A, O’Riordan WD et al : Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 379 : 11–21, 2018 – reference: 3) Yamashita T, Ueda M, Misumi Y et al : Genetic and clinical characteristics of hereditary transthyretin amyloidosis in endemic and non–endemic areas : experience from a singlereferral center in Japan. J Neurol 265 : 134–140, 2018 |
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| SubjectTerms | amyloidosis gene silencing therapy liver transplantation transthyretin TTR stabilizer |
| Title | 遺伝性トランスサイレチンアミロイドーシス治療の現状と展望 |
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