Literature DB >> 25233993

mRNA-based therapeutics--developing a new class of drugs.

Ugur Sahin1, Katalin Karikó2, Özlem Türeci3.   

Abstract

In vitro transcribed (IVT) mRNA has recently come into focus as a potential new drug class to deliver genetic information. Such synthetic mRNA can be engineered to transiently express proteins by structurally resembling natural mRNA. Advances in addressing the inherent challenges of this drug class, particularly related to controlling the translational efficacy and immunogenicity of the IVTmRNA, provide the basis for a broad range of potential applications. mRNA-based cancer immunotherapies and infectious disease vaccines have entered clinical development. Meanwhile, emerging novel approaches include in vivo delivery of IVT mRNA to replace or supplement proteins, IVT mRNA-based generation of pluripotent stem cells and genome engineering using IVT mRNA-encoded designer nucleases. This Review provides a comprehensive overview of the current state of mRNA-based drug technologies and their applications, and discusses the key challenges and opportunities in developing these into a new class of drugs.

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Year:  2014        PMID: 25233993     DOI: 10.1038/nrd4278

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN: 1474-1776            Impact factor:   84.694


  252 in total

1.  Essential role for the dsRNA-dependent protein kinase PKR in innate immunity to viral infection.

Authors:  S Balachandran; P C Roberts; L E Brown; H Truong; A K Pattnaik; D R Archer; G N Barber
Journal:  Immunity       Date:  2000-07       Impact factor: 31.745

2.  Treatment of advanced leukemia in mice with mRNA engineered T cells.

Authors:  David M Barrett; Yangbing Zhao; Xiaojun Liu; Shuguang Jiang; Carmine Carpenito; Michael Kalos; Richard G Carroll; Carl H June; Stephan A Grupp
Journal:  Hum Gene Ther       Date:  2011-09-23       Impact factor: 5.695

3.  Regimen-specific effects of RNA-modified chimeric antigen receptor T cells in mice with advanced leukemia.

Authors:  David M Barrett; Xiaojun Liu; Shuguang Jiang; Carl H June; Stephan A Grupp; Yangbing Zhao
Journal:  Hum Gene Ther       Date:  2013-08       Impact factor: 5.695

4.  Generation of gene-modified mice via Cas9/RNA-mediated gene targeting.

Authors:  Bin Shen; Jun Zhang; Hongya Wu; Jianying Wang; Ke Ma; Zheng Li; Xueguang Zhang; Pumin Zhang; Xingxu Huang
Journal:  Cell Res       Date:  2013-04-02       Impact factor: 25.617

5.  Synthetic generation of influenza vaccine viruses for rapid response to pandemics.

Authors:  Philip R Dormitzer; Pirada Suphaphiphat; Daniel G Gibson; David E Wentworth; Timothy B Stockwell; Mikkel A Algire; Nina Alperovich; Mario Barro; David M Brown; Stewart Craig; Brian M Dattilo; Evgeniya A Denisova; Ivna De Souza; Markus Eickmann; Vivien G Dugan; Annette Ferrari; Raul C Gomila; Liqun Han; Casey Judge; Sarthak Mane; Mikhail Matrosovich; Chuck Merryman; Giuseppe Palladino; Gene A Palmer; Terika Spencer; Thomas Strecker; Heidi Trusheim; Jennifer Uhlendorff; Yingxia Wen; Anthony C Yee; Jayshree Zaveri; Bin Zhou; Stephan Becker; Armen Donabedian; Peter W Mason; John I Glass; Rino Rappuoli; J Craig Venter
Journal:  Sci Transl Med       Date:  2013-05-15       Impact factor: 17.956

6.  Dendritic cells transfected with interleukin-12 and tumor-associated antigen messenger RNA induce high avidity cytotoxic T cells.

Authors:  H J Bontkes; D Kramer; J J Ruizendaal; E W M Kueter; V F I van Tendeloo; C J L M Meijer; E Hooijberg
Journal:  Gene Ther       Date:  2006-10-12       Impact factor: 5.250

7.  Activation of MDA5 requires higher-order RNA structures generated during virus infection.

Authors:  Andreas Pichlmair; Oliver Schulz; Choon-Ping Tan; Jan Rehwinkel; Hiroki Kato; Osamu Takeuchi; Shizuo Akira; Michael Way; Giampietro Schiavo; Caetano Reis e Sousa
Journal:  J Virol       Date:  2009-08-05       Impact factor: 5.103

8.  Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

Authors:  Teresa Coelho; David Adams; Ana Silva; Pierre Lozeron; Philip N Hawkins; Timothy Mant; Javier Perez; Joseph Chiesa; Steve Warrington; Elizabeth Tranter; Malathy Munisamy; Rick Falzone; Jamie Harrop; Jeffrey Cehelsky; Brian R Bettencourt; Mary Geissler; James S Butler; Alfica Sehgal; Rachel E Meyers; Qingmin Chen; Todd Borland; Renta M Hutabarat; Valerie A Clausen; Rene Alvarez; Kevin Fitzgerald; Christina Gamba-Vitalo; Saraswathy V Nochur; Akshay K Vaishnaw; Dinah W Y Sah; Jared A Gollob; Ole B Suhr
Journal:  N Engl J Med       Date:  2013-08-29       Impact factor: 91.245

9.  Hepatic failure and lactic acidosis due to fialuridine (FIAU), an investigational nucleoside analogue for chronic hepatitis B.

Authors:  R McKenzie; M W Fried; R Sallie; H Conjeevaram; A M Di Bisceglie; Y Park; B Savarese; D Kleiner; M Tsokos; C Luciano
Journal:  N Engl J Med       Date:  1995-10-26       Impact factor: 91.245

10.  Ribose 2'-O-methylation provides a molecular signature for the distinction of self and non-self mRNA dependent on the RNA sensor Mda5.

Authors:  Roland Züst; Luisa Cervantes-Barragan; Matthias Habjan; Reinhard Maier; Benjamin W Neuman; John Ziebuhr; Kristy J Szretter; Susan C Baker; Winfried Barchet; Michael S Diamond; Stuart G Siddell; Burkhard Ludewig; Volker Thiel
Journal:  Nat Immunol       Date:  2011-01-09       Impact factor: 25.606

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  453 in total

Review 1.  Progress in material design for biomedical applications.

Authors:  Mark W Tibbitt; Christopher B Rodell; Jason A Burdick; Kristi S Anseth
Journal:  Proc Natl Acad Sci U S A       Date:  2015-11-24       Impact factor: 11.205

2.  mRNA: Fulfilling the Promise of Gene Therapy.

Authors:  Drew Weissman; Katalin Karikó
Journal:  Mol Ther       Date:  2015-09       Impact factor: 11.454

3.  mRNAs biotinylated within the 5' cap and protected against decapping: new tools to capture RNA-protein complexes.

Authors:  Sylwia Bednarek; Vanesa Madan; Pawel J Sikorski; Ralf Bartenschlager; Joanna Kowalska; Jacek Jemielity
Journal:  Philos Trans R Soc Lond B Biol Sci       Date:  2018-11-05       Impact factor: 6.237

4.  Delivery of mRNA vaccine with a lipid-like material potentiates antitumor efficacy through Toll-like receptor 4 signaling.

Authors:  Hongxia Zhang; Xinru You; Xiaojuan Wang; Lei Cui; Zining Wang; Feifei Xu; Mengyun Li; Zhenggang Yang; Jinyun Liu; Peng Huang; Yang Kang; Jun Wu; Xiaojun Xia
Journal:  Proc Natl Acad Sci U S A       Date:  2021-02-09       Impact factor: 11.205

Review 5.  Advances in Biomaterials for Drug Delivery.

Authors:  Owen S Fenton; Katy N Olafson; Padmini S Pillai; Michael J Mitchell; Robert Langer
Journal:  Adv Mater       Date:  2018-05-07       Impact factor: 30.849

Review 6.  Nanoscale platforms for messenger RNA delivery.

Authors:  Bin Li; Xinfu Zhang; Yizhou Dong
Journal:  Wiley Interdiscip Rev Nanomed Nanobiotechnol       Date:  2018-05-04

7.  BNT162b2 vaccine induces neutralizing antibodies and poly-specific T cells in humans.

Authors:  Ugur Sahin; Alexander Muik; Isabel Vogler; Evelyna Derhovanessian; Lena M Kranz; Mathias Vormehr; Jasmin Quandt; Nicole Bidmon; Alexander Ulges; Alina Baum; Kristen E Pascal; Daniel Maurus; Sebastian Brachtendorf; Verena Lörks; Julian Sikorski; Peter Koch; Rolf Hilker; Dirk Becker; Ann-Kathrin Eller; Jan Grützner; Manuel Tonigold; Carsten Boesler; Corinna Rosenbaum; Ludwig Heesen; Marie-Cristine Kühnle; Asaf Poran; Jesse Z Dong; Ulrich Luxemburger; Alexandra Kemmer-Brück; David Langer; Martin Bexon; Stefanie Bolte; Tania Palanche; Armin Schultz; Sybille Baumann; Azita J Mahiny; Gábor Boros; Jonas Reinholz; Gábor T Szabó; Katalin Karikó; Pei-Yong Shi; Camila Fontes-Garfias; John L Perez; Mark Cutler; David Cooper; Christos A Kyratsous; Philip R Dormitzer; Kathrin U Jansen; Özlem Türeci
Journal:  Nature       Date:  2021-05-27       Impact factor: 49.962

Review 8.  Bispecific antibodies: Potential immunotherapies for HIV treatment.

Authors:  Giulia Fabozzi; Amarendra Pegu; Richard A Koup; Constantinos Petrovas
Journal:  Methods       Date:  2018-10-22       Impact factor: 3.608

9.  Universal Dengue Vaccine Elicits Neutralizing Antibodies against Strains from All Four Dengue Virus Serotypes.

Authors:  Naoko Uno; Ted M Ross
Journal:  J Virol       Date:  2021-01-28       Impact factor: 5.103

10.  Biomaterials for mRNA delivery.

Authors:  Mohammad Ariful Islam; Emma K G Reesor; Yingjie Xu; Harshal R Zope; Bruce R Zetter; Jinjun Shi
Journal:  Biomater Sci       Date:  2015-08-17       Impact factor: 6.843

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