Oligonucleotide Drugs for Transthyretin Amyloidosis

被引：31

作者：

Buxbaum, Joel N. ^{[1
]}

机构：

[1] Scripps Res Inst, La Jolla, CA 92037 USA

来源：

NEW ENGLAND JOURNAL OF MEDICINE | 2018年 / 379卷 / 01期

关键词：

ANTISENSE OLIGONUCLEOTIDES; POLYNEUROPATHY; TRIALS;

D O I：

10.1056/NEJMe1805499

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

[No abstract available]

引用

页码：82 / 85

页数：4

共 10 条

[1] Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides [J].

Ackermann, Elizabeth J. ;

Guo, Shuling ;

Benson, Merrill D. ;

Booten, Sheri ;

Freier, Sue ;

Hughes, Steven G. ;

Kim, Tae-Won ;

Kwoh, T. Jesse ;

Matson, John ;

Norris, Dan ;

Yu, Rosie ;

Watt, Andy ;

Monia, Brett P. .

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2016, 23 (03) :148-157

[2] Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis [J].

Adams, D. ;

Gonzalez-Duarte, A. ;

O'Riordan, W. D. ;

Yang, C. -C. ;

Ueda, M. ;

Kristen, A. V. ;

Tournev, I. ;

Schmidt, H. H. ;

Coelho, T. ;

Berk, J. L. ;

Lin, K. -P. ;

Vita, G. ;

Attarian, S. ;

Plante-Bordeneuve, V. ;

Mezei, M. M. ;

Campistol, J. M. ;

Buades, J. ;

Brannagan, T. H., III ;

Kim, B. J. ;

Oh, J. ;

Parman, Y. ;

Sekijima, Y. ;

Hawkins, P. N. ;

Solomon, S. D. ;

Polydefkis, M. ;

Dyck, P. J. ;

Gandhi, P. J. ;

Goyal, S. ;

Chen, J. ;

Strahs, A. L. ;

Nochur, S. V. ;

Sweetser, M. T. ;

Garg, P. P. ;

Vaishnaw, A. K. ;

Gollob, J. A. ;

Suhr, O. B. .

NEW ENGLAND JOURNAL OF MEDICINE, 2018, 379 (01) :11-21

[3] Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis [J].

Benson, M. D. ;

Waddington-Cruz, M. ;

Berk, J. L. ;

Polydefkis, M. ;

Dyck, P. J. ;

Wang, A. K. ;

Plante-Bordeneuve, V. ;

Barroso, F. A. ;

Merlini, G. ;

Obici, L. ;

Scheinberg, M. ;

Brannagan, T. H., III ;

Litchy, W. J. ;

Whelan, C. ;

Drachman, B. M. ;

Adams, D. ;

Heitner, S. B. ;

Conceicao, I. ;

Schmidt, H. H. ;

Vita, G. ;

Campistol, J. M. ;

Gamez, J. ;

Gorevic, P. D. ;

Gane, E. ;

Shah, A. M. ;

Solomon, S. D. ;

Monia, B. P. ;

Hughes, S. G. ;

Kwoh, T. J. ;

McEvoy, B. W. ;

Jung, S. W. ;

Baker, B. F. ;

Ackermann, E. J. ;

Gertz, M. A. ;

Coelho, T. .

NEW ENGLAND JOURNAL OF MEDICINE, 2018, 379 (01) :22-31

[4] Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial [J].

Berk, John L. ;

Suhr, Ole B. ;

Obici, Laura ;

Sekijima, Yoshiki ;

Zeldenrust, Steven R. ;

Yamashita, Taro ;

Heneghan, Michael A. ;

Gorevic, Peter D. ;

Litchy, William J. ;

Wiesman, Janice F. ;

Nordh, Erik ;

Corato, Manuel ;

Lozza, Alessandro ;

Cortese, Andrea ;

Robinson-Papp, Jessica ;

Colton, Theodore ;

Rybin, Denis V. ;

Bisbee, Alice B. ;

Ando, Yukio ;

Ikeda, Shu-ichi ;

Seldin, David C. ;

Merlini, Giampaolo ;

Skinner, Martha ;

Kelly, Jeffery W. ;

Dyck, Peter J. .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2013, 310 (24) :2658-2667

[5] Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis [J].

Coelho, Teresa ;

Adams, David ;

Silva, Ana ;

Lozeron, Pierre ;

Hawkins, Philip N. ;

Mant, Timothy ;

Perez, Javier ;

Chiesa, Joseph ;

Warrington, Steve ;

Tranter, Elizabeth ;

Munisamy, Malathy ;

Falzone, Rick ;

Harrop, Jamie ;

Cehelsky, Jeffrey ;

Bettencourt, Brian R. ;

Geissler, Mary ;

Butler, James S. ;

Sehgal, Alfica ;

Meyers, Rachel E. ;

Chen, Qingmin ;

Borland, Todd ;

Hutabarat, Renta M. ;

Clausen, Valerie A. ;

Alvarez, Rene ;

Fitzgerald, Kevin ;

Gamba-Vitalo, Christina ;

Nochur, Saraswathy V. ;

Vaishnaw, Akshay K. ;

Sah, Dinah W. Y. ;

Gollob, Jared A. ;

Suhr, Ole B. .

NEW ENGLAND JOURNAL OF MEDICINE, 2013, 369 (09) :819-829

[6] Tafamidis for transthyretin familial amyloid polyneuropathy A randomized, controlled trial [J].

Coelho, Teresa ;

Maia, Luis F. ;

da Silva, Ana Martins ;

Cruz, Marcia Waddington ;

Plante-Bordeneuve, Violaine ;

Lozeron, Pierre ;

Suhr, Ole B. ;

Campistol, Josep M. ;

Conceicao, Isabel Maria ;

Schmidt, Hartmut H. -J. ;

Trigo, Pedro ;

Kelly, Jeffery W. ;

Labaudinie, Richard ;

Chan, Jason ;

Packman, Jeff ;

Wilson, Amy ;

Grogan, Donna R. .

NEUROLOGY, 2012, 79 (08) :785-792

[7] The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials [J].

Crooke, Stanley T. ;

Baker, Brenda F. ;

Witztum, Joseph L. ;

Kwoh, Jesse ;

Pham, Nguyen C. ;

Salgado, Nelson ;

McEvoy, Bradley W. ;

Cheng, Wei ;

Hughes, Steven G. ;

Bhanot, Sanjay ;

Geary, Richard S. .

NUCLEIC ACID THERAPEUTICS, 2017, 27 (03) :121-129

[8] Liver Transplantation for Hereditary Transthyretin Amyloidosis: After 20 Years Still the Best Therapeutic Alternative? [J].

Ericzon, Bo-Goran ;

Wilczek, Henryk E. ;

Larsson, Marie ;

Wijayatunga, Priyantha ;

Stangou, Arie ;

Pena, Joao Rodrigues ;

Furtado, Emanuel ;

Barroso, Eduardo ;

Daniel, Jorge ;

Samuel, Didier ;

Adam, Rene ;

Karam, Vincent ;

Poterucha, John ;

Lewis, David ;

Ferraz-Neto, Ben-Hur ;

Cruz, Marcia Waddington ;

Munar-Ques, Miguel ;

Fabregat, Juan ;

Ikeda, Shu-ichi ;

Ando, Yukio ;

Heaton, Nigel ;

Otto, Gerd ;

Suhr, Ole .

TRANSPLANTATION, 2015, 99 (09) :1847-1854

[9] The Transthyretin Amyloidoses: From Delineating the Molecular Mechanism of Aggregation Linked to Pathology to a Regulatory-Agency-Approved Drug [J].

Johnson, Steven M. ;

Connelly, Stephen ;

Fearns, Colleen ;

Powers, Evan T. ;

Kelly, Jeffery W. .

JOURNAL OF MOLECULAR BIOLOGY, 2012, 421 (2-3) :185-203

[10] Time for one-person trials [J].

Schork, Nicholas J. .

NATURE, 2015, 520 (7549) :609-611

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