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 Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

165 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date 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. Among authors: valles a. 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 Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes.
Keskin S, Brouwers CC, Sogorb-Gonzalez M, Martier R, Depla JA, Vallès A, van Deventer SJ, Konstantinova P, Evers MM. Keskin S, et al. Among authors: valles a. Mol Ther Methods Clin Dev. 2019 Oct 4;15:275-284. doi: 10.1016/j.omtm.2019.09.010. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31737741 Free PMC article.
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology.
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M. Spronck EA, et al. Among authors: valles a. Brain Sci. 2021 Jan 20;11(2):129. doi: 10.3390/brainsci11020129. Brain Sci. 2021. PMID: 33498212 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. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system.
Sogorb-Gonzalez M, Vendrell-Tornero C, Snapper J, Stam A, Keskin S, Miniarikova J, Spronck EA, de Haan M, Nieuwland R, Konstantinova P, van Deventer SJ, Evers MM, Vallès A. Sogorb-Gonzalez M, et al. Among authors: valles a. Brain Commun. 2021 Apr 1;3(2):fcab054. doi: 10.1093/braincomms/fcab054. eCollection 2021. Brain Commun. 2021. PMID: 34704020 Free PMC article.
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.
Nobre RJ, Lobo DD, Henriques C, Duarte SP, Lopes SM, Silva AC, Lopes MM, Mariet F, Schwarz LK, Baatje MS, Ferreira V, Vallès A, Pereira de Almeida L, Evers MM, Toonen LJA. Nobre RJ, et al. Among authors: valles a. Nucleic Acid Ther. 2022 Jun;32(3):194-205. doi: 10.1089/nat.2021.0020. Epub 2021 Dec 7. Nucleic Acid Ther. 2022. PMID: 34878314 Free PMC article.
165 results