Cargando…

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

Huntington disease (HD) is a fatal neurodegenerative disease caused by a pathogenic expansion of a CAG repeat in the huntingtin (HTT) gene. There are no disease-modifying therapies for HD. Artificial microRNAs targeting HTT transcripts for degradation have shown preclinical promise and will soon ent...

Descripción completa

Detalles Bibliográficos
Autores principales:	Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2020
Materias:	Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7145682/ https://www.ncbi.nlm.nih.gov/pubmed/31745548 http://dx.doi.org/10.1093/nar/gkz976

Ejemplares similares

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models
por: Spronck, Elisabeth A., et al.
Publicado: (2019)

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes
por: Keskin, Sonay, et al.
Publicado: (2019)

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model
por: Evers, Melvin M., et al.
Publicado: (2018)

AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease
por: Miniarikova, J, et al.
Publicado: (2017)

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease
por: Miniarikova, Jana, et al.
Publicado: (2016)

Altering cortical input unmasks synaptic phenotypes in the YAC128 cortico-striatal co-culture model of Huntington disease
por: Schmidt, Mandi E., et al.
Publicado: (2018)

Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression
por: Southwell, Amber L., et al.
Publicado: (2015)

Emerging Therapies for Huntington’s Disease – Focus on N-Terminal Huntingtin and Huntingtin Exon 1
por: van der Bent, M Leontien, et al.
Publicado: (2022)

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
por: Spronck, Elisabeth A., et al.
Publicado: (2021)

DAPK1 Promotes Extrasynaptic GluN2B Phosphorylation and Striatal Spine Instability in the YAC128 Mouse Model of Huntington Disease
por: Schmidt, Mandi E., et al.
Publicado: (2020)

Conformations of Flanking Bases in HIV-1 RNA DIS Kissing Complexes Studied by Molecular Dynamics
por: Réblová, Kamila, et al.
Publicado: (2007)

Components of variance in transcriptomics based on electrophoretic separation of cDNA fragments (cDNA-AFLP)
por: Weiberg, Arne, et al.
Publicado: (2009)

RNA pseudoknots
por: Westhof, Eric, et al.
Publicado: (1992)

Isometric artifacts from polymerase chain reaction‐massively parallel sequencing analysis of short tandem repeat loci: An emerging issue from a new technology?
por: Zupanič Pajnič, Irena, et al.
Publicado: (2022)

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain
por: Depla, Josse A., et al.
Publicado: (2020)

Making (anti-) sense out of huntingtin levels in Huntington disease
por: Evers, Melvin M, et al.
Publicado: (2015)

Mechanisms of covalent self-assembly of the Azoarcus ribozyme from four fragment oligonucleotides
por: Draper, Will E., et al.
Publicado: (2008)

Cooperative motion of a key positively charged residue and metal ions for DNA replication catalyzed by human DNA Polymerase-η
por: Genna, Vito, et al.
Publicado: (2016)

Contributions of the two accessory subunits, RNASEH2B and RNASEH2C, to the activity and properties of the human RNase H2 complex
por: Chon, Hyongi, et al.
Publicado: (2009)

Wild-type huntingtin ameliorates striatal neuronal atrophy but does not prevent other abnormalities in the YAC128 mouse model of Huntington disease
por: Van Raamsdonk, Jeremy M, et al.
Publicado: (2006)

Striatal Mutant Huntingtin Protein Levels Decline with Age in Homozygous Huntington’s Disease Knock-In Mouse Models
por: Franich, Nicholas R., et al.
Publicado: (2018)

Preventing mutant huntingtin proteolysis and intermittent fasting promote autophagy in models of Huntington disease
por: Ehrnhoefer, Dagmar E., et al.
Publicado: (2018)

Striatal Synaptic Dysfunction and Hippocampal Plasticity Deficits in the Hu97/18 Mouse Model of Huntington Disease
por: Kolodziejczyk, Karolina, et al.
Publicado: (2014)

Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease
por: Martier, Raygene, et al.
Publicado: (2019)

Loss of the thyroid hormone-binding protein Crym renders striatal neurons more vulnerable to mutant huntingtin in Huntington's disease
por: Francelle, Laetitia, et al.
Publicado: (2015)

In vivo expression of polyglutamine-expanded huntingtin by mouse striatal astrocytes impairs glutamate transport: a correlation with Huntington's disease subjects
por: Faideau, Mathilde, et al.
Publicado: (2010)

Huntingtin Aggregates in the Olfactory Bulb in Huntington’s Disease
por: Highet, Blake, et al.
Publicado: (2020)

Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA
por: Donadon, Irving, et al.
Publicado: (2019)

Magnesium-binding studies reveal fundamental differences between closely related RNA triphosphatases
por: Soulière, Marie F., et al.
Publicado: (2008)

Cordycepin-hypersensitive growth links elevated polyphosphate levels to inhibition of poly(A) polymerase in Saccharomyces cerevisiae
por: Holbein, Sandra, et al.
Publicado: (2008)

The Leu22Pro tumor-associated variant of DNA polymerase beta is dRP lyase deficient
por: Dalal, Shibani, et al.
Publicado: (2008)

DNA polymerase kappa produces interrupted mutations and displays polar pausing within mononucleotide microsatellite sequences
por: Hile, Suzanne E., et al.
Publicado: (2008)

The N-terminal domain of TWINKLE contributes to single-stranded DNA binding and DNA helicase activities
por: Farge, Géraldine, et al.
Publicado: (2008)

The yeast ribosome synthesis factor Emg1 is a novel member of the superfamily of alpha/beta knot fold methyltransferases
por: Leulliot, Nicolas, et al.
Publicado: (2008)

Structure–function analysis of the RNA polymerase cleft loops elucidates initial transcription, DNA unwinding and RNA displacement
por: Naji, Souad, et al.
Publicado: (2008)

Site specific phosphorylation of yeast RNA polymerase I
por: Gerber, Jochen, et al.
Publicado: (2008)

HU binds and folds single-stranded DNA
por: Kamashev, Dmitri, et al.
Publicado: (2008)

Uracil recognition by replicative DNA polymerases is limited to the archaea, not occurring with bacteria and eukarya
por: Wardle, Josephine, et al.
Publicado: (2008)

Identification and characterization of coumestans as novel HCV NS5B polymerase inhibitors
por: Kaushik-Basu, Neerja, et al.
Publicado: (2008)

Substitution of a residue contacting the triphosphate moiety of the incoming nucleotide increases the fidelity of yeast DNA polymerase ζ
por: Howell, Craig A., et al.
Publicado: (2008)

Cannot write session to /tmp/vufind_sessions/sess_not399fujfgget5lu4ndppmtku