scholarly journals Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene

2003 ◽  
Vol 5 (6) ◽  
pp. 518-527 ◽  
Author(s):  
Stephen J. Errington ◽  
Christopher J. Mann ◽  
Sue Fletcher ◽  
Stephen D. Wilton
Keyword(s):  
Antisense Oligonucleotide ◽  
Target Selection ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Selection For

scholarly journals Antisense oligonucleotide modified with serinol nucleic acid (SNA) induces exon skipping in mdx myotubes

RSC Advances ◽  
2017 ◽  
Vol 7 (54) ◽  
pp. 34049-34052 ◽  
Author(s):  
Bao T. Le ◽  
Keiji Murayama ◽  
Fazel Shabanpoor ◽  
Hiroyuki Asanuma ◽  
Rakesh N. Veedu
Keyword(s):  
Nucleic Acid ◽  
Antisense Oligonucleotide ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Gene Transcript

We investigated the potential of SNA-modified antisense oligonucleotide (AO) for exon-skipping. We found that a 20-mer SNA-AO induced efficient exon-23 skipping in the mouse dystrophin gene transcript.

scholarly journals Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries

2007 ◽  
Vol 8 (1) ◽  
pp. 57 ◽  
Author(s):  
Abbie M Adams ◽  
Penny L Harding ◽  
Patrick L Iversen ◽  
Catherine Coleman ◽  
Sue Fletcher ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Gene Transcript

scholarly journals Guanine Analogues Enhance Antisense Oligonucleotide-induced Exon Skipping in Dystrophin Gene In Vitro and In Vivo

2010 ◽  
Vol 18 (4) ◽  
pp. 812-818 ◽  
Author(s):  
Yihong Hu ◽  
Bo Wu ◽  
Allen Zillmer ◽  
Peijuan Lu ◽  
Ehsan Benrashid ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Exon Skipping ◽  
Dystrophin Gene

scholarly journals Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript

2007 ◽  
Vol 15 (7) ◽  
pp. 1288-1296 ◽  
Author(s):  
Steve D Wilton ◽  
Abbie M Fall ◽  
Penny L Harding ◽  
Graham McClorey ◽  
Catherine Coleman ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Gene Transcript ◽  
Human Dystrophin

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

2015 ◽  
Vol 15 (6) ◽  
pp. 563-571 ◽  
Author(s):  
Mirella Meregalli ◽  
Andrea Farini ◽  
Clementina Sitzia ◽  
Cyriaque Beley ◽  
Paola Razini ◽  
...  
Keyword(s):  
Stem Cell ◽  
Bystander Effect ◽  
Exon Skipping ◽  
Dystrophin Gene

Track Segment Association in Target Selection for Interdiction Using a Single Passive Sensor

2021 ◽  
pp. 1-1
Author(s):  
K. Yang ◽  
M. Danino ◽  
Y. Bar-Shalom ◽  
D. Belfadel ◽  
B. Milgrom ◽  
...  
Keyword(s):  
Target Selection ◽  
Track Segment ◽  
Passive Sensor ◽  
Selection For

scholarly journals Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy

2021 ◽  
Author(s):  
Vratko Himič ◽  
Kay E. Davies
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Structural Integrity ◽  
Viral Vector ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Pharmacological Treatments ◽  
Genetic Sequencing ◽  
Reading Frame ◽  
Target Effects

AbstractDuchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the structural integrity of muscle. As variants in the dystrophin gene lead to a disruption of the reading frame, pharmacological treatments have only limited efficacy; there is currently no effective therapy and consequently, a significant unmet clinical need for DMD. Recently, novel genetic approaches have shown real promise in treating DMD, with advancements in the efficacy and tropism of exon skipping and surrogate gene therapy. CRISPR-Cas9 has the potential to be a ‘one-hit’ curative treatment in the coming decade. The current limitations of gene editing, such as off-target effects and immunogenicity, are in fact partly constraints of the delivery method itself, and thus research focus has shifted to improving the viral vector. In order to halt the loss of ambulation, early diagnosis and treatment will be pivotal. In an era where genetic sequencing is increasingly utilised in the clinic, genetic therapies will play a progressively central role in DMD therapy. This review delineates the relative merits of cutting-edge genetic approaches, as well as the challenges that still need to be overcome before they become clinically viable.

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