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The use of genetic tools in experimental therapy of polyglutamine diseases.

2015/18/E/NZ2/00678

Keywords

Descriptors

  • NZ2_1: Genetyka molekularna
  • NZ5_7: Leczenie chorób człowieka
  • NZ1_1: Biologia molekularna

Panel

NZ2 – Genetics, genomics: molecular genetics, genomics, proteomics, bioinformatics, systems biology, genetic epidemiology

Host institution

Instytut Chemii Bioorganicznej PAN

Voivodeship: wielkopolskie

Other projects carried out by the institution »

Principal investigator (from the host institution)

dr hab. Marta Olejniczak

Number of co-investigators in the project: 4

Call

SONATA BIS 5 – announced on 2015-06-15

Amount awarded
1 917 600 PLN
Project start date
2016-04-18
Project end date
2021-10-17
Project duration
66 months (the same as in the proposal)
Project status
Project settled

Project description

Download the project description in a pdf file

Note — project descriptions were prepared by the authors of the applications themselves and placed in the system in an unchanged form.

Equipment purchased [PL]

  1. Inkubator bakteryjny z wyposażeniem.
  2. aparat do elektroforezy i transferu białek z zasilaczem. Cost: 30 000 PLN
  3. aparat do elektroforezy westem blot dedykowany do rozdzialu bialek w komercyjnych żelach gradientowych.
  4. licznik komórek. Cost: 15 000 PLN
  5. termocykler. Cost: 30 000 PLN
  6. zestaw adaptujący wieszak MTP do rotora wirówki.
  7. zestaw komputerowy wraz z oprogramowaniem (2 pcs). Cost: 9 000 PLN

Information in the final report

Publication in academic press/journals (9)

  1. Artificial miRNAs as therapeutic tools: challenges and opportunities

    Authors:
    Kotowska-Zimmer A, Pewinska M, Olejniczak M
    Academic press:
    WIREs RNA (year: 2021, volume: nie dotyczy, pages: nie dotyczy), Publisher: Wiley Periodicals LLC
    Status:
    Published
    DOI:
    10.1002/wrna.1640 – link to the publication

  2. Paving the way towards precise and safe CRISPR genome editing

    Authors:
    Pawel Sledzinski, Magdalena Dabrowska, Mateusz Nowaczyk, Marta Olejniczak
    Academic press:
    Biotechnology Advances (year: 2021, volume: 49, pages: 46042), Publisher: Elsevier
    Status:
    Published
    DOI:
    10.1016/j.biotechadv.2021.107737 – link to the publication

  3. Generation of new isogenic models of Huntington's disease using CRISPR-Cas9 technology

    Authors:
    Dabrowska M, Ciolak A, Kozlowska E, Fiszer A. and Olejniczak M
    Academic press:
    International Journal of Molecular Sciences (year: 2020, volume: 21(5), pages: 1854), Publisher: MDPI
    Status:
    Published
    DOI:
    10.3390/ijms21051854 – link to the publication

  4. qEva-CRISPR: a method for quantitative evaluation of CRISPR/Cas-mediated genome editing in target and off-target sites

    Authors:
    Dabrowska M, Czubak K, Juzwa W, Krzyzosiak WJ, Olejniczak M, Kozlowski P
    Academic press:
    Nucleic Acids Res (year: 2018, volume: 46(17), pages: e101), Publisher: Oxford Academic
    Status:
    Published
    DOI:
    10.1093/nar/gky505 – link to the publication

  5. Stress-induced changes in miRNA biogenesis and functioning

    Authors:
    Olejniczak M, Kotowska-Zimmer A, Krzyzosiak WJ
    Academic press:
    Cell Mol Life Sci., (year: 2018, volume: 75, pages: 177-191), Publisher: Springer
    Status:
    Published
    DOI:
    10.1007/s00018-017-2591-0 – link to the publication

  6. Precise excision of the CAG tract from the Huntingtin Gene by Cas9 Nickases

    Authors:
    Dabrowska M, Juzwa W, Krzyzosiak WJ, Olejniczak M
    Academic press:
    Front Neurosci (year: 2018, volume: 0,552083333333333, pages: 46030), Publisher: Frontiers Media SA
    Status:
    Published
    DOI:
    10.3389/fnins.2018.00075 – link to the publication

  7. Generation of human iPS cell line IBCHi001-A from dentatorubral-pallidoluysian atrophy patient's fibroblasts

    Authors:
    Kozlowska E, Ciolak A, Olejniczak M, Fiszer A.
    Academic press:
    Stem Cell Research (year: 2019, volume: 39, pages: 101512), Publisher: Elsevier
    Status:
    Published
    DOI:
    10.1016/j.scr.2019.101512 – link to the publication

  8. Universal RNAi Triggers for the Specific Inhibition of Mutant Huntingtin, Atrophin-1, Ataxin-3, and Ataxin-7 Expression

    Authors:
    Kotowska-Zimmer A, Ostrovska Y, Olejniczak M
    Academic press:
    Mol Ther Nucleic Acids (year: 2020, volume: 19, pages: 562-571), Publisher: CellPress
    Status:
    Published
    DOI:
    10.1016/j.omtn.2019.12.012 – link to the publication

  9. A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease

    Authors:
    Kotowska-Zimmer A., Przybyl L., Pewinska M., Suszynska-Zajczyk J., Wronka D., Figiel M. and Olejniczak M
    Status:
    Accepted for publication

Articles in post-conference publications (2)

  1. Universal RNAi triggers for specific inhibition of mutant huntingtin, ataxin-3, ataxin-7 and atrophin-1 expression

    Authors:
    A. Kotowska-Zimmer, J. Ostrowska, K. Grochowina, A. Ciesiołka, P. Joachimiak, A. Fiszer, M. Olejniczak
    Conference:
    the 44th FEBS Congress (year: 2019, pages: 51), Publisher: FEBS Open Bio
    Date:
    conference 6-11.07.2019
    Status:
    Published

  2. A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease

    Authors:
    M. Olejniczak, A. Kotowska-Zimmer, L. Przybyl, M. Pewińska, J. Suszynska-Zajczyk, D. Wronka, M. Figiel
    Conference:
    European Huntington's Disease Network (EHDN) Meeting (year: 2021, pages: nd), Publisher: BMJ Journals
    Date:
    conference 9-11.09
    Status:
    Published

Book publications / chapters in book publications (1)

  1. Gene Therapy for Huntington's Disease Using Targeted Endonucleases

    Authors:
    Magdalena Dabrowska and Marta Olejniczak
    Book:
    Methods in Molecular Biology, Trinucleotide repeats (year: 2020, volume: 2056, pages: 269-284), Publisher: Humana Press
    Status:
    Published

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