Search Result "Delivery of exon skipping machinery."


Personalised Genetic Intervention for Duchenne Muscular Dystrophy: Antisense Oligomers and Exon Skipping

Journal: Current Molecular Pharmacology
Volume: 2 Issue: 1 Year: 2009 Page: 110-121
Author(s): Chalermchai Mitrpant, Sue Fletcher, Steve D. Wilton

Stem Cell-Mediated Exon Skipping of the Dystrophin Gene by the Bystander Effect

Journal: Current Gene Therapy
Volume: 15 Issue: 6 Year: 2015 Page: 563-571
Author(s): Mirella Meregalli,Andrea Farini,Clementina Sitzia,Cyriaque Beley,Paola Razini,Letizia Cassinelli,Federica Colleoni,Paola Frattini,Nadia Santo,Elisabetta Galbiati,Davide Prosperi,Alessandro Tavelli,Marzia Belicchi,Luis Garcia,Yvan Torrente

Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)

Journal: Current Gene Therapy
Volume: 15 Issue: 4 Year: 2015 Page: 395-415
Author(s): Klaudia Kawecka,Michael Theodoulides,Yalin Hasoglu,Susan Jarmin,Hanna Kymalainen,Anita Le-Heron,Linda Popplewell,Alberto Malerba,George Dickson,Takis Athanasopoulos

Developing Effective Therapy for Duchenne Muscular Dystrophy (DMD): Challenges and Promises

Journal: Current Chemical Biology
Volume: 8 Issue: 3 Year: 2014 Page: 117-131
Author(s): Trinath Ghosh,Ujjal Ghosh,Gargi Ghosh,Dabasish Malik,Shankhamala Bose,Utpal Basu

Splicing Modulation Mediated by Small Nuclear RNAs as Therapeutic Approaches for Muscular Dystrophies

Journal: Current Gene Therapy
Volume: 12 Issue: 3 Year: 2012 Page: 179-191
Author(s): Rachid Benchaouir,Aurelie Goyenvalle

Peptide-Mediated Cellular Delivery of Oligonucleotide-Based Therapeutics In Vitro: Quantitative Evaluation of Overall Efficacy Employing Easy to Handle Reporter Systems

Journal: Current Pharmaceutical Design
Volume: 14 Issue: 3 Year: 2008 Page: 3637-3655
Author(s): S. D. Laufer, T. Restle

RNA Splicing Manipulation: Strategies to Modify Gene Expression for a Variety of Therapeutic Outcomes

Journal: Current Gene Therapy
Volume: 11 Issue: 4 Year: 2011 Page: 259-275
Author(s): Steve D. Wilton, Susan Fletcher

Cell-penetrating Peptides as Nucleic Acid Delivery Systems: From Biophysics to Biological Applications

Journal: Current Pharmaceutical Design
Volume: 19 Issue: 1 Year: 2013 Page: 2895-2923
Author(s): Sara Trabulo, Ana L. Cardoso, Ana M. S. Cardoso, Catarina M. Morais, Amalia S. Jurado, Maria C. Pedroso de Lima

Gene Targeting and Expression Modulation by Peptide Nucleic Acids (PNA)

Journal: Current Pharmaceutical Design
Volume: 16 Issue: 2 Year: 2010 Page: 3118-3123
Author(s): Peter E. Nielsen

RNA Splicing Manipulation: Strategies to Modify Gene Expression for a Variety of Therapeutic Outcomes

Journal: Current Gene Therapy
Volume: 5 Issue: 5 Year: 2005 Page: 467-483
Author(s): Steve D. Wilton, Susan Fletcher

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