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 Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy

2018 ◽  
Vol 8 (4) ◽  
pp. 38 ◽  
Author(s):  
Kenji Lim ◽  
Chantal Yoon ◽  
Toshifumi Yokota
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Wide Spectrum ◽  
Membrane Integrity ◽  
Dystrophin Gene ◽  
Muscle Membrane ◽  
Reading Frame ◽  
Long Term Treatment

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disease prevalent in 1 in 3500 to 5000 males worldwide. As a result of mutations that interrupt the reading frame of the dystrophin gene (DMD), DMD is characterized by a loss of dystrophin protein that leads to decreased muscle membrane integrity, which increases susceptibility to degeneration. CRISPR/Cas9 technology has garnered interest as an avenue for DMD therapy due to its potential for permanent exon skipping, which can restore the disrupted DMD reading frame in DMD and lead to dystrophin restoration. An RNA-guided DNA endonuclease system, CRISPR/Cas9 allows for the targeted editing of specific sequences in the genome. The efficacy and safety of CRISPR/Cas9 as a therapy for DMD has been evaluated by numerous studies in vitro and in vivo, with varying rates of success. Despite the potential of CRISPR/Cas9-mediated gene editing for the long-term treatment of DMD, its translation into the clinic is currently challenged by issues such as off-targeting, immune response activation, and sub-optimal in vivo delivery. Its nature as being mostly a personalized form of therapy also limits applicability to DMD patients, who exhibit a wide spectrum of mutations. This review summarizes the various CRISPR/Cas9 strategies that have been tested in vitro and in vivo for the treatment of DMD. Perspectives on the approach will be provided, and the challenges faced by CRISPR/Cas9 in its road to the clinic will be briefly discussed.

The uptake of a fluorescently labelled antisense oligonucleotide in vitro and in vivo

2005 ◽  
Vol 147 (1) ◽  
pp. 48-54 ◽  
Author(s):  
Emma S.J. Robinson ◽  
David J. Nutt ◽  
Helen C. Jackson ◽  
Alan L. Hudson
Keyword(s):  
Antisense Oligonucleotide

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 Magnetofection—A highly efficient tool for antisense oligonucleotide delivery in vitro and in vivo

2003 ◽  
Vol 7 (5) ◽  
pp. 700-710 ◽  
Author(s):  
Florian Krötz ◽  
Cor de Wit ◽  
Hae-Young Sohn ◽  
Stefan Zahler ◽  
Torsten Gloe ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Efficient Tool ◽  
Highly Efficient ◽  
Oligonucleotide Delivery

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 In vivo recognition of a vertebrate mini-exon as an exon-intron-exon unit.

1993 ◽  
Vol 13 (5) ◽  
pp. 2677-2687 ◽  
Author(s):  
D A Sterner ◽  
S M Berget
Keyword(s):  
Splice Site ◽  
Rna Splicing ◽  
Troponin I ◽  
Exon Skipping ◽  
Splice Sites ◽  
Small Vertebrate ◽  
Upstream Exon ◽  
I Gene

Very small vertebrate exons are problematic for RNA splicing because of the proximity of their 3' and 5' splice sites. In this study, we investigated the recognition of a constitutive 7-nucleotide mini-exon from the troponin I gene that resides quite close to the adjacent upstream exon. The mini-exon failed to be included in spliced RNA when placed in a heterologous gene unless accompanied by the upstream exon. The requirement for the upstream exon disappeared when the mini-exon was internally expanded, suggesting that the splice sites bordering the mini-exon are compatible with those of other constitutive vertebrate exons and that the small size of the exon impaired inclusion. Mutation of the 5' splice site of the natural upstream exon did not result in either exon skipping or activation of a cryptic 5' splice site, the normal vertebrate phenotypes for such mutants. Instead, a spliced RNA accumulated that still contained the upstream intron. In vitro, the mini-exon failed to assemble into spliceosome complexes unless either internally expanded or accompanied by the upstream exon. Thus, impaired usage of the mini-exon in vivo was accompanied by impaired recognition in vitro, and recognition of the mini-exon was facilitated by the presence of the upstream exon in vivo and in vitro. Cumulatively, the atypical in vivo and in vitro properties of the troponin exons suggest a mechanism for the recognition of this mini-exon in which initial recognition of an exon-intron-exon unit is followed by subsequent recognition of the intron.

An anionic long-circulating liposome that improves radioiodinated antisense oligonucleotide delivery in vitro and in vivo

2011 ◽  
Vol 31 (1) ◽  
pp. 20-28 ◽  
Author(s):  
Jiawei Xie ◽  
Chao Ma ◽  
Jizheng Lin ◽  
Guoming Wang ◽  
Anren Kuang ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Oligonucleotide Delivery
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