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  1. AU="Shcherbinina, Evgeniya Y"
  2. AU="Rothfuss, Katja"
  3. AU="Resmana Lim"

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  1. Article ; Online: Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications.

    Sergeeva, Olga V / Shcherbinina, Evgeniya Y / Shomron, Noam / Zatsepin, Timofei S

    Nucleic acid therapeutics

    2022  Volume 32, Issue 3, Page(s) 123–138

    Abstract: Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic approaches have been developed to correct aberrant alternative splicing events for the treatment of cancers and hereditary diseases, including gene therapy and ... ...

    Abstract Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic approaches have been developed to correct aberrant alternative splicing events for the treatment of cancers and hereditary diseases, including gene therapy and redirecting splicing, using small molecules or splice switching oligonucleotides (SSO). Significant advances in the chemistry and pharmacology of nucleic acid have led to the development of clinically approved SSO drugs for the treatment of spinal muscular dystrophy and Duchenne muscular dystrophy (DMD). In this review, we discuss the mechanisms of SSO action with emphasis on "less common" approaches to modulate alternative splicing, including bipartite and bifunctional SSO, oligonucleotide decoys for splice factors and SSO-mediated mRNA degradation
    MeSH term(s) Alternative Splicing/genetics ; Humans ; Muscular Dystrophy, Duchenne/drug therapy ; Muscular Dystrophy, Duchenne/genetics ; Oligonucleotides/genetics ; Oligonucleotides/pharmacology ; Oligonucleotides/therapeutic use ; Oligonucleotides, Antisense/genetics ; Oligonucleotides, Antisense/metabolism ; Oligonucleotides, Antisense/therapeutic use ; RNA Splicing/genetics
    Chemical Substances Oligonucleotides ; Oligonucleotides, Antisense
    Language English
    Publishing date 2022-02-14
    Publishing country United States
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 2639888-6
    ISSN 2159-3345 ; 2159-3337
    ISSN (online) 2159-3345
    ISSN 2159-3337
    DOI 10.1089/nat.2021.0067
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: lncRNA in the liver: Prospects for fundamental research and therapy by RNA interference.

    Smekalova, Elena M / Kotelevtsev, Yuri V / Leboeuf, Dominique / Shcherbinina, Evgeniya Y / Fefilova, Anna S / Zatsepin, Timofei S / Koteliansky, Victor

    Biochimie

    2016  Volume 131, Page(s) 159–172

    Abstract: Long non-coding RNAs constitute the most abundant part of the transcribed mammalian genome. lncRNAs affect all essential processes in the living cell including transcription, splicing, translation, replication, shaping of chromatin and post translational ...

    Abstract Long non-coding RNAs constitute the most abundant part of the transcribed mammalian genome. lncRNAs affect all essential processes in the living cell including transcription, splicing, translation, replication, shaping of chromatin and post translational modification of proteins. Alterations in lncRNA expression have been linked to a number of diseases; thus, modulation of lncRNA expression holds a huge potential for gene-based therapy. In this review we summarize published data about lncRNAs in the context of hepatic carcinogenesis and liver fibrosis, and the corresponding potential targets for gene therapy. Recent advancements in targeted delivery to the liver made RNA interference an invaluable tool to decipher hepatic lncRNA function and to develop lncRNA-oriented therapies for liver-involved diseases in the future. Different approaches for RNA delivery that can be used for functional studies in the lab and for clinical lncRNA based applications are critically discussed in this review.
    MeSH term(s) Animals ; Biomedical Research/methods ; Gene Expression Regulation ; Genetic Therapy/methods ; Humans ; Liver/metabolism ; Liver/pathology ; Liver Cirrhosis/genetics ; Liver Cirrhosis/therapy ; Liver Neoplasms/genetics ; Liver Neoplasms/therapy ; RNA Interference ; RNA, Long Noncoding/genetics
    Chemical Substances RNA, Long Noncoding
    Language English
    Publishing date 2016-12
    Publishing country France
    Document type Journal Article ; Review
    ZDB-ID 120345-9
    ISSN 1638-6183 ; 0300-9084
    ISSN (online) 1638-6183
    ISSN 0300-9084
    DOI 10.1016/j.biochi.2016.06.007
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article: lncRNA in the liver: Prospects for fundamental research and therapy by RNA interference

    Smekalova, Elena M / Kotelevtsev, Yuri V / Leboeuf, Dominique / Shcherbinina, Evgeniya Y / Fefilova, Anna S / Zatsepin, Timofei S / Kotelianski, Victor

    Biochimie. 2016,

    2016  

    Abstract: Long non-coding RNAs constitute the most abundant part of the transcribed mammalian genome. lncRNAs affect all essential processes in the living cell including transcription, splicing, translation, replication, shaping of chromatin and post translational ...

    Abstract Long non-coding RNAs constitute the most abundant part of the transcribed mammalian genome. lncRNAs affect all essential processes in the living cell including transcription, splicing, translation, replication, shaping of chromatin and post translational modification of proteins. Alterations in lncRNA expression have been linked to a number of diseases; thus, modulation of lncRNA expression holds a huge potential for gene-based therapy. In this review we summarize published data about lncRNAs in the context of hepatic carcinogenesis and liver fibrosis, and the corresponding potential targets for gene therapy. Recent advancements in targeted delivery to the liver made RNA interference an invaluable tool to decipher hepatic lncRNA function and to develop lncRNA-oriented therapies for liver-involved diseases in the future. Different approaches for RNA delivery that can be used for functional studies in the lab and for clinical lncRNA based applications are critically discussed in this review.
    Keywords RNA interference ; carcinogenesis ; chromatin ; gene therapy ; genome ; liver ; liver cirrhosis ; mammals ; non-coding RNA ; transcription (genetics) ; translation (genetics)
    Language English
    Size p. .
    Publishing place Elsevier B.V.
    Document type Article
    Note Pre-press version
    ZDB-ID 120345-9
    ISSN 0300-9084
    ISSN 0300-9084
    DOI 10.1016/j.biochi.2016.06.007
    Database NAL-Catalogue (AGRICOLA)

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