Literature DB >> 31537746

Messenger RNA therapy as an option for treating metabolic disorders.

Randy J Chandler1.   

Abstract

Entities:  

Year:  2019        PMID: 31537746      PMCID: PMC6800380          DOI: 10.1073/pnas.1914673116

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN: 0027-8424            Impact factor:   11.205


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  11 in total

Review 1.  A New Era for Rare Genetic Diseases: Messenger RNA Therapy.

Authors:  Paolo G V Martini; Lin T Guey
Journal:  Hum Gene Ther       Date:  2019-07-01       Impact factor: 5.695

2.  Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency.

Authors:  Mary G Prieve; Pierrot Harvie; Sean D Monahan; Debashish Roy; Allen G Li; Teri L Blevins; Amber E Paschal; Matt Waldheim; Eric C Bell; Anna Galperin; Jean-Rene Ella-Menye; Michael E Houston
Journal:  Mol Ther       Date:  2018-01-04       Impact factor: 11.454

3.  Arginase I mRNA therapy - a novel approach to rescue arginase 1 enzyme deficiency.

Authors:  Kirtika H Asrani; Lei Cheng; Christopher J Cheng; Romesh R Subramanian
Journal:  RNA Biol       Date:  2018-07-24       Impact factor: 4.652

4.  Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.

Authors:  Ding An; Jessica L Schneller; Andrea Frassetto; Shi Liang; Xuling Zhu; Ji-Sun Park; Matt Theisen; Sue-Jean Hong; Jenny Zhou; Raj Rajendran; Becca Levy; Rebecca Howell; Gilles Besin; Vladimir Presnyak; Staci Sabnis; Kerry E Murphy-Benenato; E Sathyajith Kumarasinghe; Timothy Salerno; Cosmin Mihai; Christine M Lukacs; Randy J Chandler; Lin T Guey; Charles P Venditti; Paolo G V Martini
Journal:  Cell Rep       Date:  2017-12-19       Impact factor: 9.423

5.  Long-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.

Authors:  Eun K Lee; Chuhong Hu; Ragini Bhargava; Nora Rozengurt; David Stout; Wayne W Grody; Stephen D Cederbaum; Gerald S Lipshutz
Journal:  Mol Ther       Date:  2012-07-03       Impact factor: 11.454

6.  mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency.

Authors:  Jingsong Cao; Ding An; Mikel Galduroz; Jenny Zhuo; Shi Liang; Marianne Eybye; Andrea Frassetto; Eishi Kuroda; Aki Funahashi; Jordan Santana; Cosmin Mihai; Kerry E Benenato; E Sathyajith Kumarasinghe; Staci Sabnis; Timothy Salerno; Kimberly Coughlan; Edward J Miracco; Becca Levy; Gilles Besin; Joshua Schultz; Christine Lukacs; Lin Guey; Patrick Finn; Tatsuhiko Furukawa; Paloma H Giangrande; Takeyori Saheki; Paolo G V Martini
Journal:  Mol Ther       Date:  2019-04-23       Impact factor: 11.454

7.  Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency.

Authors:  Brian Truong; Gabriella Allegri; Xiao-Bo Liu; Kristine E Burke; Xuling Zhu; Stephen D Cederbaum; Johannes Häberle; Paolo G V Martini; Gerald S Lipshutz
Journal:  Proc Natl Acad Sci U S A       Date:  2019-09-09       Impact factor: 11.205

8.  Systemic messenger RNA as an etiological treatment for acute intermittent porphyria.

Authors:  Lei Jiang; Pedro Berraondo; Daniel Jericó; Lin T Guey; Ana Sampedro; Andrea Frassetto; Kerry E Benenato; Kristine Burke; Eva Santamaría; Manuel Alegre; Álvaro Pejenaute; Mayur Kalariya; William Butcher; Ji-Sun Park; Xuling Zhu; Staci Sabnis; E Sathyajith Kumarasinghe; Timothy Salerno; Matthew Kenney; Christine M Lukacs; Matías A Ávila; Paolo G V Martini; Antonio Fontanellas
Journal:  Nat Med       Date:  2018-10-08       Impact factor: 53.440

9.  Human recombinant arginase enzyme reduces plasma arginine in mouse models of arginase deficiency.

Authors:  Lindsay C Burrage; Qin Sun; Sarah H Elsea; Ming-Ming Jiang; Sandesh C S Nagamani; Arthur E Frankel; Everett Stone; Susan E Alters; Dale E Johnson; Scott W Rowlinson; George Georgiou; Brendan H Lee
Journal:  Hum Mol Genet       Date:  2015-09-10       Impact factor: 6.150

10.  Gene Therapy for Metabolic Diseases.

Authors:  Randy J Chandler; Charles P Venditti
Journal:  Transl Sci Rare Dis       Date:  2016
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  4 in total

1.  Challenges and Opportunities for Nucleic Acid Therapeutics.

Authors:  David R Corey; Masad J Damha; Muthiah Manoharan
Journal:  Nucleic Acid Ther       Date:  2021-12-17       Impact factor: 5.486

Review 2.  The legacy of mRNA engineering: A lineup of pioneers for the Nobel Prize.

Authors:  Miroslaw Janowski; Anna Andrzejewska
Journal:  Mol Ther Nucleic Acids       Date:  2022-07-13       Impact factor: 10.183

3.  Subcutaneous delivery of FGF21 mRNA therapy reverses obesity, insulin resistance, and hepatic steatosis in diet-induced obese mice.

Authors:  Stefano Bartesaghi; Kristina Wallenius; Daniel Hovdal; Mathias Liljeblad; Simonetta Wallin; Niek Dekker; Louise Barlind; Nigel Davies; Frank Seeliger; Maria Sörhede Winzell; Sima Patel; Matt Theisen; Luis Brito; Nils Bergenhem; Shalini Andersson; Xiao-Rong Peng
Journal:  Mol Ther Nucleic Acids       Date:  2022-04-18       Impact factor: 10.183

Review 4.  The Pivotal Role of Chemical Modifications in mRNA Therapeutics.

Authors:  Albert Liu; Xiao Wang
Journal:  Front Cell Dev Biol       Date:  2022-07-13
  4 in total

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