Literature DB >> 29860876

The macrophage as a Trojan horse for antisense oligonucleotide delivery.

James S Novak1,2,3, Jyoti K Jaiswal1,2,3, Terence A Partridge1,2,3.   

Abstract

Entities:  

Keywords:  Duchenne muscular dystrophy; antisense oligonucleotides; drug delivery; exon skipping; macrophages; satellite cells

Mesh：

Substances：

Year:  2018        PMID： 29860876      PMCID： PMC6309535          DOI： 10.1080/14728222.2018.1482279

Source DB:  PubMed          Journal:  Expert Opin Ther Targets        ISSN： 1472-8222            Impact factor:   6.902


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  18 in total

1.  Eteplirsen for the treatment of Duchenne muscular dystrophy.

Authors:  Jerry R Mendell; Louise R Rodino-Klapac; Zarife Sahenk; Kandice Roush; Loren Bird; Linda P Lowes; Lindsay Alfano; Ann Maria Gomez; Sarah Lewis; Janaiah Kota; Vinod Malik; Kim Shontz; Christopher M Walker; Kevin M Flanigan; Marco Corridore; John R Kean; Hugh D Allen; Chris Shilling; Kathleen R Melia; Peter Sazani; Jay B Saoud; Edward M Kaye
Journal:  Ann Neurol       Date:  2013-09-10       Impact factor: 10.422

2.  Feasibility of SPECT-CT Imaging to Study the Pharmacokinetics of Antisense Oligonucleotides in a Mouse Model of Duchenne Muscular Dystrophy.

Authors:  Evita van de Steeg; Tilman Läppchen; Begoña Aguilera; Harm T Jansen; Daan Muilwijk; Rick Vermue; José W van der Hoorn; Katia Donato; Raffaella Rossin; Peter C de Visser; Maria L H Vlaming
Journal:  Nucleic Acid Ther       Date:  2017-04-18       Impact factor: 5.486

3.  Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.

Authors:  Sebahattin Cirak; Virginia Arechavala-Gomeza; Michela Guglieri; Lucy Feng; Silvia Torelli; Karen Anthony; Stephen Abbs; Maria Elena Garralda; John Bourke; Dominic J Wells; George Dickson; Matthew J A Wood; Steve D Wilton; Volker Straub; Ryszard Kole; Stephen B Shrewsbury; Caroline Sewry; Jennifer E Morgan; Kate Bushby; Francesco Muntoni
Journal:  Lancet       Date:  2011-07-23       Impact factor: 79.321

4.  Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate.

Authors:  Adams Amantana; Hong M Moulton; Melissa L Cate; Muralimohan T Reddy; Tom Whitehead; Jed N Hassinger; Derek S Youngblood; Patrick L Iversen
Journal:  Bioconjug Chem       Date:  2007-06-21       Impact factor: 4.774

Review 5.  Antisense oligonucleotides: the next frontier for treatment of neurological disorders.

Authors:  Carlo Rinaldi; Matthew J A Wood
Journal:  Nat Rev Neurol       Date:  2017-12-01       Impact factor: 42.937

6.  Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.

Authors:  Aurélie Goyenvalle; Graziella Griffith; Arran Babbs; Samir El Andaloussi; Kariem Ezzat; Aurélie Avril; Branislav Dugovic; Rémi Chaussenot; Arnaud Ferry; Thomas Voit; Helge Amthor; Claudia Bühr; Stefan Schürch; Matthew J A Wood; Kay E Davies; Cyrille Vaillend; Christian Leumann; Luis Garcia
Journal:  Nat Med       Date:  2015-02-02       Impact factor: 53.440

7.  Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.

Authors:  James S Novak; Marshall W Hogarth; Jessica F Boehler; Marie Nearing; Maria C Vila; Raul Heredia; Alyson A Fiorillo; Aiping Zhang; Yetrib Hathout; Eric P Hoffman; Jyoti K Jaiswal; Kanneboyina Nagaraju; Sebahattin Cirak; Terence A Partridge
Journal:  Nat Commun       Date:  2017-10-16       Impact factor: 14.919

8.  Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.

Authors:  Taavi Lehto; Alejandra Castillo Alvarez; Sarah Gauck; Michael J Gait; Thibault Coursindel; Matthew J A Wood; Bernard Lebleu; Prisca Boisguerin
Journal:  Nucleic Acids Res       Date:  2013-12-23       Impact factor: 16.971

9.  Elusive sources of variability of dystrophin rescue by exon skipping.

Authors:  Maria Candida Vila; Margaret Benny Klimek; James S Novak; Sree Rayavarapu; Kitipong Uaesoontrachoon; Jessica F Boehler; Alyson A Fiorillo; Marshall W Hogarth; Aiping Zhang; Conner Shaughnessy; Heather Gordish-Dressman; Umar Burki; Volker Straub; Qi Long Lu; Terence A Partridge; Kristy J Brown; Yetrib Hathout; John van den Anker; Eric P Hoffman; Kanneboyina Nagaraju
Journal:  Skelet Muscle       Date:  2015-12-01       Impact factor: 4.912

Review 10.  Delivery is key: lessons learnt from developing splice-switching antisense therapies.

Authors:  Caroline Godfrey; Lourdes R Desviat; Bård Smedsrød; France Piétri-Rouxel; Michela A Denti; Petra Disterer; Stéphanie Lorain; Gisela Nogales-Gadea; Valentina Sardone; Rayan Anwar; Samir El Andaloussi; Taavi Lehto; Bernard Khoo; Camilla Brolin; Willeke Mc van Roon-Mom; Aurélie Goyenvalle; Annemieke Aartsma-Rus; Virginia Arechavala-Gomeza
Journal:  EMBO Mol Med       Date:  2017-05       Impact factor: 12.137
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  3 in total

1.  PMO-based let-7c site blocking oligonucleotide (SBO) mediated utrophin upregulation in mdx mice, a therapeutic approach for Duchenne muscular dystrophy (DMD).

Authors:  Kasturi Sengupta; Emanuele Loro; Tejvir S Khurana
Journal:  Sci Rep       Date:  2020-12-09       Impact factor: 4.379

Review 2.  Cell-derived membrane biomimetic nanocarriers for targeted therapy of pulmonary disease.

Authors:  Xixi Zheng; Tianyuan Zhang; Ting Huang; Yanjun Zhou; Jianqing Gao
Journal:  Int J Pharm       Date:  2022-04-18       Impact factor: 6.510

3.  Connexin-43 reduction prevents muscle defects in a mouse model of manifesting Duchenne muscular dystrophy female carriers.

Authors:  Julie Nouet; Eric Himelman; Kevin C Lahey; Qingshi Zhao; Diego Fraidenraich
Journal:  Sci Rep       Date:  2020-03-30       Impact factor: 4.379
  3 in total

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