Zobrazeno 1 - 10
of 247
pro vyhledávání: '"Steve D. Wilton"'
Publikováno v:
Frontiers in Molecular Neuroscience, Vol 17 (2024)
Down syndrome is a genetic-based disorder that results from the triplication of chromosome 21, leading to an overexpression of many triplicated genes, including the gene encoding Dual-Specificity Tyrosine Phosphorylation-Regulated Kinase 1A (DYRK1A).
Externí odkaz:
https://doaj.org/article/7bd9e0f2f9304a299516f800d438406f
Autor:
Jessica M. Cale, Kristin A. Ham, Dunhui Li, Craig S. McIntosh, Gerald F. Watts, Steve D. Wilton, May T. Aung-Htut
Publikováno v:
Scientific Reports, Vol 13, Iss 1, Pp 1-11 (2023)
Abstract Splice modulating antisense oligomers (AOs) are increasingly used to modulate RNA processing. While most are investigated for their use as therapeutics, AOs can also be used for basic research. This study examined their use to investigate in
Externí odkaz:
https://doaj.org/article/10bd2f65b9ba4a9785fccfd552be3f14
Autor:
Kristin A. Ham, Niall P. Keegan, Craig S. McIntosh, May T. Aung-Htut, Khine Zaw, Kane Greer, Sue Fletcher, Steve D. Wilton
Publikováno v:
Scientific Reports, Vol 11, Iss 1, Pp 1-13 (2021)
Abstract Antisense oligomers (AOs) are increasingly being used to modulate RNA splicing in live cells, both for research and for the development of therapeutics. While the most common intended effect of these AOs is to induce skipping of whole exons,
Externí odkaz:
https://doaj.org/article/5312637696694b37aa54f9c7ed7d275a
Publikováno v:
Frontiers in Genetics, Vol 13 (2022)
Externí odkaz:
https://doaj.org/article/69bdad9e8dbe4f62a1217208267b5403
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 22, Iss , Pp 263-272 (2020)
Dystrophin plays a crucial role in maintaining sarcolemma stability during muscle contractions, and mutations that prevent the expression of a functional protein cause Duchenne muscular dystrophy (DMD). Antisense oligonucleotide-mediated manipulation
Externí odkaz:
https://doaj.org/article/5194b4eb8cc142648cdf40c35c8296d1
Autor:
Loren L. Flynn, Ruohan Li, Ianthe L. Pitout, May T. Aung-Htut, Leon M. Larcher, Jack A. L. Cooper, Kane L. Greer, Alysia Hubbard, Lisa Griffiths, Charles S. Bond, Steve D. Wilton, Archa H. Fox, Sue Fletcher
Publikováno v:
Frontiers in Genetics, Vol 13 (2022)
Oligonucleotides and nucleic acid analogues that alter gene expression are now showing therapeutic promise in human disease. Whilst the modification of synthetic nucleic acids to protect against nuclease degradation and to influence drug function is
Externí odkaz:
https://doaj.org/article/ad5ce073129249a4a67d5806834bf299
Autor:
Kelly M. Martinovich, Anthony Kicic, Stephen M. Stick, Russell D. Johnsen, Sue Fletcher, Steve D. Wilton
Publikováno v:
Frontiers in Pharmacology, Vol 13 (2022)
Introduction: Severity and disease progression in people with Cystic Fibrosis (CF) is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides (AO).
Externí odkaz:
https://doaj.org/article/a0021259ab664828891fea13507550e8
Publikováno v:
Frontiers in Genetics, Vol 12 (2022)
Understanding pre-mRNA splicing is crucial to accurately diagnosing and treating genetic diseases. However, mutations that alter splicing can exert highly diverse effects. Of all the known types of splicing mutations, perhaps the rarest and most diff
Externí odkaz:
https://doaj.org/article/8539cadce7004dbf8d01f49e526ad9c5
Autor:
Di Huang, Dan Zhang, Shang-Chih Chen, May Thandar Aung-Htut, Tina M. Lamey, Jennifer A. Thompson, Terri L. McLaren, John N. De Roach, Sue Fletcher, Steve D. Wilton, Samuel McLenachan, Fred K. Chen
Publikováno v:
Stem Cell Research, Vol 54, Iss , Pp 102439- (2021)
Mutations in ABCA4 gene are causative for autosomal recessive Stargardt disease (STGD1), the most common inherited retinal dystrophy. Here, we report the generation of an induced pluripotent stem cell (iPSC) line from a STGD1 patient carrying biallel
Externí odkaz:
https://doaj.org/article/f673ade02e254d86b1205023ee61cecb
Autor:
Bao T. Le, Prithi Raguraman, Tamer R. Kosbar, Susan Fletcher, Steve D. Wilton, Rakesh N. Veedu
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 14, Iss , Pp 142-157 (2019)
Cancer is one of the leading causes of death worldwide, and conventional cancer therapies such as surgery, chemotherapy, and radiotherapy do not address the underlying molecular pathologies, leading to inadequate treatment and tumor recurrence. Angio
Externí odkaz:
https://doaj.org/article/23cc5a31134b4538b0c334e998770fc2