612 related articles for article (PubMed ID: 30007561)
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
Mol Ther; 2018 Sep; 26(9):2163-2177. PubMed ID: 30007561
[TBL] [Abstract][Full Text] [Related]
2. AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington's disease mouse model.
Thomson SB; Stam A; Brouwers C; Fodale V; Bresciani A; Vermeulen M; Mostafavi S; Petkau TL; Hill A; Yung A; Russell-Schulz B; Kozlowski P; MacKay A; Ma D; Beg MF; Evers MM; Vallès A; Leavitt BR
Brain; 2023 Jun; 146(6):2298-2315. PubMed ID: 36508327
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. 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]
5. Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study.
Morais RDVS; Sogorb-González M; Bar C; Timmer NC; Van der Bent ML; Wartel M; Vallès A
Cells; 2022 Sep; 11(17):. PubMed ID: 36078156
[TBL] [Abstract][Full Text] [Related]
6. AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models.
Spronck EA; Brouwers CC; Vallès A; de Haan M; Petry H; van Deventer SJ; Konstantinova P; Evers MM
Mol Ther Methods Clin Dev; 2019 Jun; 13():334-343. PubMed ID: 30984798
[TBL] [Abstract][Full Text] [Related]
7. 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
Sci Transl Med; 2021 Apr; 13(588):. PubMed ID: 33827977
[TBL] [Abstract][Full Text] [Related]
8. 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]
9. 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
Brain Sci; 2021 Jan; 11(2):. PubMed ID: 33498212
[TBL] [Abstract][Full Text] [Related]
10. 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
Mol Ther Methods Clin Dev; 2019 Dec; 15():275-284. PubMed ID: 31737741
[TBL] [Abstract][Full Text] [Related]
11. 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]
12. 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]
13. A transgenic minipig model of Huntington's Disease.
Baxa M; Hruska-Plochan M; Juhas S; Vodicka P; Pavlok A; Juhasova J; Miyanohara A; Nejime T; Klima J; Macakova M; Marsala S; Weiss A; Kubickova S; Musilova P; Vrtel R; Sontag EM; Thompson LM; Schier J; Hansikova H; Howland DS; Cattaneo E; DiFiglia M; Marsala M; Motlik J
J Huntingtons Dis; 2013; 2(1):47-68. PubMed ID: 25063429
[TBL] [Abstract][Full Text] [Related]
14. 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]
15. Minipigs as a Large-Brained Animal Model for Huntington's Disease: From Behavior and Imaging to Gene Therapy.
Reilmann R; Schuldenzucker V
Methods Mol Biol; 2018; 1780():241-266. PubMed ID: 29856023
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Gradual Phenotype Development in Huntington Disease Transgenic Minipig Model at 24 Months of Age.
Vidinská D; Vochozková P; Šmatlíková P; Ardan T; Klíma J; Juhás Š; Juhásová J; Bohuslavová B; Baxa M; Valeková I; Motlík J; Ellederová Z
Neurodegener Dis; 2018; 18(2-3):107-119. PubMed ID: 29870995
[TBL] [Abstract][Full Text] [Related]
18. Brain urea increase is an early Huntington's disease pathogenic event observed in a prodromal transgenic sheep model and HD cases.
Handley RR; Reid SJ; Brauning R; Maclean P; Mears ER; Fourie I; Patassini S; Cooper GJS; Rudiger SR; McLaughlan CJ; Verma PJ; Gusella JF; MacDonald ME; Waldvogel HJ; Bawden CS; Faull RLM; Snell RG
Proc Natl Acad Sci U S A; 2017 Dec; 114(52):E11293-E11302. PubMed ID: 29229845
[TBL] [Abstract][Full Text] [Related]
19. [Gene silencing approaches for the treatment of Huntington's disease].
Merienne N; Déglon N
Med Sci (Paris); 2015 Feb; 31(2):159-67. PubMed ID: 25744262
[TBL] [Abstract][Full Text] [Related]
20. AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease.
Franich NR; Fitzsimons HL; Fong DM; Klugmann M; During MJ; Young D
Mol Ther; 2008 May; 16(5):947-56. PubMed ID: 18388917
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]