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A biological classification of Huntington's disease: the Integrated Staging System.
Tabrizi SJ, Schobel S, Gantman EC, Mansbach A, Borowsky B, Konstantinova P, Mestre TA, Panagoulias J, Ross CA, Zauderer M, Mullin AP, Romero K, Sivakumaran S, Turner EC, Long JD, Sampaio C; Huntington's Disease Regulatory Science Consortium (HD-RSC). Tabrizi SJ, et al. Among authors: konstantinova p. Lancet Neurol. 2022 Jul;21(7):632-644. doi: 10.1016/S1474-4422(22)00120-X. Lancet Neurol. 2022. PMID: 35716693 Review.
Therapeutic efficacy of regulable GDNF expression for Huntington's and Parkinson's disease by a high-induction, background-free "GeneSwitch" vector.
Cheng S, Tereshchenko J, Zimmer V, Vachey G, Pythoud C, Rey M, Liefhebber J, Raina A, Streit F, Mazur A, Bähr M, Konstantinova P, Déglon N, Kügler S. Cheng S, et al. Among authors: konstantinova p. Exp Neurol. 2018 Nov;309:79-90. doi: 10.1016/j.expneurol.2018.07.017. Epub 2018 Aug 1. Exp Neurol. 2018. PMID: 30076831
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. Miniarikova J, et al. Among authors: konstantinova p. Gene Ther. 2017 Oct;24(10):630-639. doi: 10.1038/gt.2017.71. Epub 2017 Aug 3. Gene Ther. 2017. PMID: 28771234 Free PMC article.
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. Caron NS, et al. Among authors: konstantinova p. Nucleic Acids Res. 2020 Jan 10;48(1):36-54. doi: 10.1093/nar/gkz976. Nucleic Acids Res. 2020. PMID: 31745548 Free PMC article.
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. Evers MM, et al. Among authors: konstantinova p. Mol Ther. 2018 Sep 5;26(9):2163-2177. doi: 10.1016/j.ymthe.2018.06.021. Epub 2018 Jun 25. Mol Ther. 2018. PMID: 30007561 Free PMC article.
Widespread and sustained target engagement in Huntington's disease minipigs upon intrastriatal microRNA-based gene therapy.
Vallès A, Evers MM, Stam A, Sogorb-Gonzalez M, Brouwers C, Vendrell-Tornero C, Acar-Broekmans S, Paerels L, Klima J, Bohuslavova B, Pintauro R, Fodale V, Bresciani A, Liscak R, Urgosik D, Starek Z, Crha M, Blits B, Petry H, Ellederova Z, Motlik J, van Deventer S, Konstantinova P. Vallès A, et al. Among authors: konstantinova p. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver.
Maczuga P, Verheij J, van der Loos C, van Logtenstein R, Hooijer G, Martier R, Borel F, Lubelski J, Koornneef A, Blits B, van Deventer S, Petry H, Konstantinova P. Maczuga P, et al. Among authors: konstantinova p. Gene Ther. 2014 Jan;21(1):60-70. doi: 10.1038/gt.2013.58. Epub 2013 Oct 24. Gene Ther. 2014. PMID: 24152580 Free PMC article.
Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery.
Pietersz KL, Martier RM, Baatje MS, Liefhebber JM, Brouwers CC, Pouw SM, Fokkert L, Lubelski J, Petry H, Martens GJM, van Deventer SJ, Konstantinova P, Blits B. Pietersz KL, et al. Among authors: konstantinova p. Gene Ther. 2021 Aug;28(7-8):435-446. doi: 10.1038/s41434-020-0178-0. Epub 2020 Aug 15. Gene Ther. 2021. PMID: 32801344
44 results