Q84328 (Q84328): Difference between revisions
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(Created claim: summary (P836): Myotonic Dystrophy (DM) and Fragile X-associated tremor-Ataxia Syndrome (FXTAS) are dominant disorders caused by RNA gain of function of expanded CUG or CGG repeats. Both RNAs may sequester nuclear proteins, and their repeat sequences Undergo untypical translation into toxic mono-amino acid peptides. Since the causative molecular target is well defined, DM and FXTAS are highly amenable to the development of RNA targeting therapy. We and others d...) |
(Changed label, description and/or aliases in 1 language: remove_english_label) |
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Revision as of 12:36, 14 October 2020
Project in Poland financed by DG Regio
| Language | Label | Description | Also known as |
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| English | No label defined |
Project in Poland financed by DG Regio |
Statements
3,499,222.0 zloty
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3,499,222.0 zloty
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100.0 percent
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1 October 2018
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30 September 2021
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UNIWERSYTET IM. ADAMA MICKIEWICZA W POZNANIU
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Q2513981 (Deleted Item)
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Myotonic Dystrophy (DM) and Fragile X-associated tremor-Ataxia Syndrome (FXTAS) are dominant disorders caused by RNA gain of function of expanded CUG or CGG repeats. Both RNAs may sequester nuclear proteins, and their repeat sequences undergo untypical translation into toxic mono-amino acid peptides. Since the causative molecular target is well defined, DM and FXTAS are highly amenable to the development of RNA targeting therapy. We and others demonstrated that reduction of protein sequestration on toxic CUG repeats using various oligonucleotide-based approaches or small compounds led to promising results in animal models of DM; therefore, now we want to develop similar tools to reduce the effect of FXTAS mutation by targeting RNA with CGG expansion, to reduce its translation rate. We also aim to achieve the gene therapy tool to overexpress the therapeutic proteins which may rescue nuclear proteins from pathogenic sequestration in DM. We are going to study mechanisms of both diseases. (Polish)
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Myotonic Dystrophy (DM) and Fragile X-associated tremor-Ataxia Syndrome (FXTAS) are dominant disorders caused by RNA gain of function of expanded CUG or CGG repeats. Both RNAs may sequester nuclear proteins, and their repeat sequences Undergo untypical translation into toxic mono-amino acid peptides. Since the causative molecular target is well defined, DM and FXTAS are highly amenable to the development of RNA targeting therapy. We and others demonstrated that reduction of protein sequestration on toxic CUG repeats using various oligonucleotide-based approaches or small compounds led to promising results in animal models of DM; therefore, now we want to develop similar tools to reduce the effect of FXTAS mutation by targeting RNA with CGG expansion, to reduce its translation rate. We also aim to achieve the gene therapy tool to overexpress the therapeutic proteins which may rescue nuclear proteins from pathogenic sequestration in DM. We are going to study mechanisms of both diseases. (English)
14 October 2020
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Identifiers
POIR.04.04.00-00-5C0C/17
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