The use of chemically modified antisense oligonucleotides in the experimental therapy against Myotonic Dystrophy (DM).
2014/15/B/NZ5/00142
Keywords
Descriptors
- NZ1_1: Biologia molekularna
- NZ2_1: Genetyka molekularna
- NZ5_3: Patogeneza chorób człowieka
Panel
NZ5 – Human and animal noninfectious diseases: etiology, mechanisms, diagnosis and treatment of diseases, poisonings and injuries (without neurological diseases)
Host institution
Uniwersytet im. Adama Mickiewicza w Poznaniu, Wydział Biologii
Voivodeship: wielkopolskie
Principal investigator (from the host institution)
dr Ewa Agnieszka Stepniak-Konieczna
Number of co-investigators in the project: 4
Call
OPUS 8 – announced on 2014-09-15
- Amount awarded
- 1 231 620 PLN
- Project start date
- 2015-07-23
- Project end date
- 2021-01-22
- Project duration
- 66 months (the same as in the proposal)
- Project status
- Project settled
Information in the final report
Publication in academic press/journals (4)
-
Cyclic mismatch binding ligands interact with disease-associated CGG trinucleotide repeats in RNA and suppress their translation
- Authors:
- Patryk Konieczny, Sanjukta Mukherjee, Ewa Stepniak-Konieczna, Katarzyna Taylor, Daria Niewiadomska, Agnieszka Piasecka, Agnieszka Walczak, Anna Baud, Chikara Dohno, Kazuhiko Nakatani, Krzysztof Sobczak
- Academic press:
- Nucleic Acids Research (year: 2021, volume: 2021, pages: 46039), Publisher: Oxford University Press
- Status:
- Published
- DOI:
- 10.1093/nar/gkab669 – link to the publication
-
MBNL expression in autoregulatory feedback loops.
- Authors:
- Patryk Konieczny, Ewa Stepniak-Konieczna, Krzysztof Sobczak
- Academic press:
- RNA Biology (year: 2018, volume: 15(1), pages: 46030), Publisher: Taylor Francis Online
- Status:
- Published
- DOI:
- 10.1080/15476286.2017.1384119. – link to the publication
-
AON-induced splice-switching and DMPK pre-mRNA degradation as potential therapeutic approaches for Myotonic Dystrophy type 1
- Authors:
- Ewa Stepniak-Konieczna, Patryk Konieczny, Piotr Cywoniuk, Julia Dluzewska, Krzysztof Sobczak
- Academic press:
- Nucleic Acids Research (year: 2020, volume: 48(5), pages: 2531–2543), Publisher: Oxford University Press
- Status:
- Published
- DOI:
- 10.1093/nar/gkaa007 – link to the publication
-
Autoregulation of MBNL1 function by exon 1 exclusion from MBNL1 transcript.
- Authors:
- Patryk Konieczny, Ewa Stepniak-Konieczna, Katarzyna Taylor, Łukasz J. Sznajder, Krzysztof Sobczak
- Academic press:
- Nucleic Acids Research (year: 2017, volume: 45 (4), pages: 1760-1775), Publisher: Oxford University Press
- Status:
- Published
- DOI:
- 10.1093/nar/gkw1158 – link to the publication
Articles in post-conference publications (2)
-
AON-induced splice-switching and DMPK transcript degradation as therapeutic approaches for Myotonic Dystrophy type 1 (DM1)
- Authors:
- Ewa Stepniak-Konieczna, Patryk Konieczny, Julia Zielinska, Krzysztof Sobczak
- Conference:
- RNA 2018 - The 23rd Annual Meeting of The RNA Society (year: 2018, pages: 694), Publisher: The RNA Society & University of California Berkeley
- Date:
- conference 29.05-03.06
- Status:
- Published
- DOI:
- link to the publication
-
Testing therapeutic potential of splice-switching antisense oligomers targeting DMPK in cell models for DM1.
- Authors:
- STEPNIAK-KONIECZNA Ewa, KONIECZNY Patryk, ZIELINSKA Julia, SOBCZAK Krzysztof
- Conference:
- IDMC-11 International Myotonic Dystrophy Consortium Meeting (year: 2017, pages: 197), Publisher: International Myotonic Dystrophy Consortium
- Date:
- conference 5-9.09.2017
- Status:
- Published
Book publications / chapters in book publications (1)
-
Modified Antisense Oligonucleotides and Their Analogs in Therapy of Neuromuscular Diseases
- Authors:
- Patryk Konieczny, Ewa Stepniak-Konieczna, Krzysztof Sobczak
- Book:
- Modified Nucleic Acids in Biology and Medicine (year: 2016, volume: RNATechnologies, pages: 243-271), Publisher: Springer International Publishing
- Status:
- Published
- DOI:
- link to the publication