Projects funded by the NCN

Information on the principal investigator and host institution

Information of the project and the call

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The use of chemically modified antisense oligonucleotides in the experimental therapy against Myotonic Dystrophy (DM).

2014/15/B/NZ5/00142

Keywords:

antisense oligonucleotides splicing RNA myotonic dystrophy experimental therapy

Descriptors:

  • NZ1_1: Molecular biology
  • NZ2_1: Molecular genetics
  • NZ5_3: Pathogenesis of human diseases

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

woj. wielkopolskie

Other projects carried out by the institution 

Principal investigator (from the host institution):

dr Ewa 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 (Y-m-d): 2015-07-23

Project end date (Y-m-d): 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)
  • Articles in post-conference publications (2)
  • Book publications / chapters in book publications (1)
  1. 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 (rok: 2017, tom: 45 (4), strony: 1760-1775), Wydawca: Oxford University Press
    Status:
    Published
    DOI:
    10.1093/nar/gkw1158 - link to the publication
  2. 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 (rok: 2021, tom: 2021, strony: 45308), Wydawca: Oxford University Press
    Status:
    Published
    DOI:
    10.1093/nar/gkab669 - link to the publication
  3. 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 (rok: 2020, tom: 48(5), strony: 2531–2543), Wydawca: Oxford University Press
    Status:
    Published
    DOI:
    10.1093/nar/gkaa007 - link to the publication
  4. MBNL expression in autoregulatory feedback loops.
    Authors:
    Patryk Konieczny, Ewa Stepniak-Konieczna, Krzysztof Sobczak
    Academic press:
    RNA Biology (rok: 2018, tom: 15(1), strony: 45299), Wydawca: Taylor Francis Online
    Status:
    Published
    DOI:
    10.1080/15476286.2017.1384119. - link to the publication
  1. 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 (rok: 2018, ), Wydawca: The RNA Society & University of California Berkeley
    Data:
    konferencja 29.05-03.06
    Status:
    Published
    DOI:
    link to the publication
  2. 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 (rok: 2017, ), Wydawca: International Myotonic Dystrophy Consortium
    Data:
    konferencja 5-9.09.2017
    Status:
    Published
  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 (rok: 2016, tom: RNATechnologies, strony: 243-271), Wydawca: Springer International Publishing
    Status:
    Published
    DOI:
    link to the publication

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