BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

196 related articles for article (PubMed ID: 34806402)

  • 1. Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy
    Wang W; Zhou P; Wang X; Chen F; Christensen E; Thompson J; Ren X; Kells A; Stanek L; Carter T; Hou J; Sah DWY
    Hum Gene Ther; 2022 Jan; 33(1-2):37-60. PubMed ID: 34806402
    [TBL] [Abstract][Full Text] [Related]  

  • 2. AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
    Evers MM; Miniarikova J; Juhas S; Vallès A; Bohuslavova B; Juhasova J; Skalnikova HK; Vodicka P; Valekova I; Brouwers C; Blits B; Lubelski J; Kovarova H; Ellederova Z; van Deventer SJ; Petry H; Motlik J; Konstantinova P
    Mol Ther; 2018 Sep; 26(9):2163-2177. PubMed ID: 30007561
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.
    Miniarikova J; Evers MM; Konstantinova P
    Mol Ther; 2018 Apr; 26(4):947-962. PubMed ID: 29503201
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
    Caron NS; Southwell AL; Brouwers CC; Cengio LD; Xie Y; Black HF; Anderson LM; Ko S; Zhu X; van Deventer SJ; Evers MM; Konstantinova P; Hayden MR
    Nucleic Acids Res; 2020 Jan; 48(1):36-54. PubMed ID: 31745548
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Cellular Analysis of Silencing the Huntington's Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice.
    Keeler AM; Sapp E; Chase K; Sottosanti E; Danielson E; Pfister E; Stoica L; DiFiglia M; Aronin N; Sena-Esteves M
    J Huntingtons Dis; 2016 Oct; 5(3):239-248. PubMed ID: 27689620
    [TBL] [Abstract][Full Text] [Related]  

  • 6. AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
    Miniarikova J; Zimmer V; Martier R; Brouwers CC; Pythoud C; Richetin K; Rey M; Lubelski J; Evers MM; van Deventer SJ; Petry H; Déglon N; Konstantinova P
    Gene Ther; 2017 Oct; 24(10):630-639. PubMed ID: 28771234
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon?
    Liu W; Pfister EL; Kennington LA; Chase KO; Mueller C; DiFiglia M; Aronin N
    J Huntingtons Dis; 2016; 5(1):33-8. PubMed ID: 27003665
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.
    Miniarikova J; Zanella I; Huseinovic A; van der Zon T; Hanemaaijer E; Martier R; Koornneef A; Southwell AL; Hayden MR; van Deventer SJ; Petry H; Konstantinova P
    Mol Ther Nucleic Acids; 2016 Mar; 5(3):e297. PubMed ID: 27003755
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Full sequence of mutant huntingtin 3'-untranslated region and modulation of its gene regulatory activity by endogenous microRNA.
    Kim KH; Abu Elneel K; Shin JW; Keum JW; Seong D; Kwak S; Lee R; Gusella JF; MacDonald ME; Seong IS; Lee JM
    J Hum Genet; 2019 Oct; 64(10):995-1004. PubMed ID: 31296921
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Artificial miRNAs as therapeutic tools: Challenges and opportunities.
    Kotowska-Zimmer A; Pewinska M; Olejniczak M
    Wiley Interdiscip Rev RNA; 2021 Jul; 12(4):e1640. PubMed ID: 33386705
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Astrocyte transduction is required for rescue of behavioral phenotypes in the YAC128 mouse model with AAV-RNAi mediated HTT lowering therapeutics.
    Stanek LM; Bu J; Shihabuddin LS
    Neurobiol Dis; 2019 Sep; 129():29-37. PubMed ID: 31042572
    [TBL] [Abstract][Full Text] [Related]  

  • 12. A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease.
    Kotowska-Zimmer A; Przybyl L; Pewinska M; Suszynska-Zajczyk J; Wronka D; Figiel M; Olejniczak M
    Mol Ther Nucleic Acids; 2022 Jun; 28():702-715. PubMed ID: 35664700
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Lowering Mutant Huntingtin Using Tricyclo-DNA Antisense Oligonucleotides As a Therapeutic Approach for Huntington's Disease.
    Imbert M; Blandel F; Leumann C; Garcia L; Goyenvalle A
    Nucleic Acid Ther; 2019 Oct; 29(5):256-265. PubMed ID: 31184975
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Alternative processing of human HTT mRNA with implications for Huntington's disease therapeutics.
    Fienko S; Landles C; Sathasivam K; McAteer SJ; Milton RE; Osborne GF; Smith EJ; Jones ST; Bondulich MK; Danby ECE; Phillips J; Taxy BA; Kordasiewicz HB; Bates GP
    Brain; 2022 Dec; 145(12):4409-4424. PubMed ID: 35793238
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks.
    Leavitt BR; Kordasiewicz HB; Schobel SA
    JAMA Neurol; 2020 Jun; 77(6):764-772. PubMed ID: 32202594
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
    Pfister EL; DiNardo N; Mondo E; Borel F; Conroy F; Fraser C; Gernoux G; Han X; Hu D; Johnson E; Kennington L; Liu P; Reid SJ; Sapp E; Vodicka P; Kuchel T; Morton AJ; Howland D; Moser R; Sena-Esteves M; Gao G; Mueller C; DiFiglia M; Aronin N
    Hum Gene Ther; 2018 Jun; 29(6):663-673. PubMed ID: 29207890
    [TBL] [Abstract][Full Text] [Related]  

  • 17. A Fresh Look at Huntingtin mRNA Processing in Huntington's Disease.
    Romo L; Mohn ES; Aronin N
    J Huntingtons Dis; 2018; 7(2):101-108. PubMed ID: 29865084
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Artificial microRNA guide strand selection from duplexes with no mismatches shows a purine-rich preference for virus- and non-virus-based expression vectors in plants.
    Kuo YW; Falk BW
    Plant Biotechnol J; 2022 Jun; 20(6):1069-1084. PubMed ID: 35113475
    [TBL] [Abstract][Full Text] [Related]  

  • 19. The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain.
    Datson NA; González-Barriga A; Kourkouta E; Weij R; van de Giessen J; Mulders S; Kontkanen O; Heikkinen T; Lehtimäki K; van Deutekom JC
    PLoS One; 2017; 12(2):e0171127. PubMed ID: 28182673
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice.
    Agustín-Pavón C; Mielcarek M; Garriga-Canut M; Isalan M
    Mol Neurodegener; 2016 Sep; 11(1):64. PubMed ID: 27600816
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 10.