[HTML][HTML] Antisense-mediated splice intervention to treat human disease: The odyssey continues
Recent approvals of oligonucleotide analogue drugs to alter gene expression have been
welcomed by patient communities but not universally supported. These compounds …
welcomed by patient communities but not universally supported. These compounds …
Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1
The COL7A1 gene encodes homotrimer fibrils essential for anchoring dermal and epidermal
layers, and pathogenic mutations in COL7A1 can cause recessive or dominant dystrophic …
layers, and pathogenic mutations in COL7A1 can cause recessive or dominant dystrophic …
Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides
MT Aung-Htut, I Comerford, R Johnsen, K Foyle… - Scientific reports, 2019 - nature.com
With recent approvals of antisense oligonucleotides as therapeutics, there is an increasing
interest in expanding the application of these compounds to many other diseases. Our …
interest in expanding the application of these compounds to many other diseases. Our …
Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies
The literature surrounding the use of antisense oligonucleotides continues to grow, with new
disease and mechanistic applications constantly evolving. Furthermore, the discovery and …
disease and mechanistic applications constantly evolving. Furthermore, the discovery and …
Removal of the polyglutamine repeat of ataxin-3 by redirecting pre-mRNA processing
Spinocerebellar ataxia type 3 (SCA3) is a devastating neurodegenerative disease for which
there is currently no cure, nor effective treatment strategy. One of nine polyglutamine …
there is currently no cure, nor effective treatment strategy. One of nine polyglutamine …
Consequences of making the inactive active through changes in antisense oligonucleotide chemistries
Antisense oligonucleotides are short, single-stranded nucleic acid analogues that can
interfere with pre-messenger RNA (pre-mRNA) processing and induce excision of a targeted …
interfere with pre-messenger RNA (pre-mRNA) processing and induce excision of a targeted …
[PDF][PDF] Antisense oligonucleotide-mediated alternative splicing strategies to treat the type-1 fibrillinopathies
JM Cale - 2021 - researchportal.murdoch.edu.au
The type-1 fibrillinopathies are a family of connective tissue disorders of which Marfan
syndrome is the most common, affecting between 2-3 in 10,000 individuals. Marfan …
syndrome is the most common, affecting between 2-3 in 10,000 individuals. Marfan …
[PDF][PDF] the odyssey continues [version 1; peer review: 3 approved]
Recent approvals of oligonucleotide analogue drugs to alter gene expression have been
welcomed by patient communities but not universally supported. These compounds …
welcomed by patient communities but not universally supported. These compounds …
[PDF][PDF] Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides
SD Wilton - 2019 - researchportal.murdoch.edu.au
Results Analysis of the ITGA4 transcript and structure. ITGA4 is a cell surface integrin
receptor protein encoded by 28 exons (Supplementary Fig. S1). The functional full-length …
receptor protein encoded by 28 exons (Supplementary Fig. S1). The functional full-length …