Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

38 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
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. 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.
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: evers mm. 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.
Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy.
Martier R, Liefhebber JM, García-Osta A, Miniarikova J, Cuadrado-Tejedor M, Espelosin M, Ursua S, Petry H, van Deventer SJ, Evers MM, Konstantinova P. Martier R, et al. Among authors: evers mm. Mol Ther Nucleic Acids. 2019 Jun 7;16:26-37. doi: 10.1016/j.omtn.2019.02.001. Epub 2019 Feb 11. Mol Ther Nucleic Acids. 2019. PMID: 30825670 Free PMC article.
Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA.
Donadon I, Bussani E, Riccardi F, Licastro D, Romano G, Pianigiani G, Pinotti M, Konstantinova P, Evers M, Lin S, Rüegg MA, Pagani F. Donadon I, et al. Nucleic Acids Res. 2019 Aug 22;47(14):7618-7632. doi: 10.1093/nar/gkz469. Nucleic Acids Res. 2019. PMID: 31127278 Free PMC article.
38 results