UB Paderborn / Katalog / Suche / in CDI

Sortiert nach

Einfacher Volltextzugriff von (fast) überall

Installieren Sie die LibKey Nomad-Browsererweiterung und erhalten Sie von vielen Webseiten einfachen Zugriff auf lizenzierte Volltexte oder Open Access Publikationen. Mehr erfahren...

Current prospects for RNA interference-based therapies

Nature reviews. Genetics, 2011-05, Vol.12 (5), p.329-340

2011

Volltextzugriff (PDF)

Reinstating aberrant mTORC1 activity in Huntington's disease mice improves disease phenotypes

Neuron (Cambridge, Mass.), 2015-01, Vol.85 (2), p.303-315

2015

Volltextzugriff (PDF)

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

Molecular therapy, 2017-01, Vol.25 (1), p.12-23

2017

Volltextzugriff (PDF)

Transcriptome-wide discovery of microRNA binding sites in human brain

Neuron (Cambridge, Mass.), 2014-01, Vol.81 (2), p.294-305

2014

Volltextzugriff (PDF)

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

Molecular therapy, 2022-08, Vol.30 (8), p.2646-2663

2022

Volltextzugriff (PDF)

The Bifunctional microRNA miR-9/miR-9 Regulates REST and CoREST and Is Downregulated in Huntington's Disease

The Journal of neuroscience, 2008-12, Vol.28 (53), p.14341-14346

2008

Volltextzugriff (PDF)

Striatal neurons directly converted from Huntington's disease patient fibroblasts recapitulate age-associated disease phenotypes

Nature neuroscience, 2018-03, Vol.21 (3), p.341-352

2018

Volltextzugriff (PDF)

DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencing

Genome Biology, 2022-04, Vol.23 (1), p.108-108, Article 108

2022

Volltextzugriff (PDF)

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

Molecular therapy, 2009-01, Vol.17 (1), p.169-175

2009

Volltextzugriff (PDF)

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

Nature Medicine, 2009-10, Vol.15 (10), p.1215-1218

2009

Volltextzugriff (PDF)

An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion

Nature communications, 2022-03, Vol.13 (1), p.1150-1150, Article 1150

2022

Volltextzugriff (PDF)

Transvascular delivery of small interfering RNA to the central nervous system

Nature, 2007-07, Vol.448 (7149), p.39-43

2007

Volltextzugriff (PDF)

Standard screening methods underreport AAV-mediated transduction and gene editing

Nature communications, 2019-07, Vol.10 (1), p.3415-10, Article 3415

2019

Volltextzugriff (PDF)

Viral Vectors for Gene Transfer

Current protocols in mouse biology, 2018-12, Vol.8 (4), p.e58-n/a

2018

Volltextzugriff (PDF)

PIAS1 Regulates Mutant Huntingtin Accumulation and Huntington’s Disease-Associated Phenotypes In Vivo

Neuron (Cambridge, Mass.), 2016-05, Vol.90 (3), p.507-520

2016

Volltextzugriff (PDF)

Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy

Molecular therapy, 2014-03, Vol.22 (3), p.588-595

2014

Volltextzugriff (PDF)

Structure and activity of putative intronic miRNA promoters

RNA (Cambridge), 2010-03, Vol.16 (3), p.495-505

2010

Volltextzugriff (PDF)

Safety and Efficacy of Ex Vivo Donor Lung Adenoviral IL-10 Gene Therapy in a Large Animal Lung Transplant Survival Model

Human gene therapy, 2017-09, Vol.28 (9), p.757-765

2017

Volltextzugriff (PDF)

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

Molecular therapy, 2009-06, Vol.17 (6), p.1053-1063

2009

Volltextzugriff (PDF)

RNAi prevents and reverses phenotypes induced by mutant human ataxin-1

Annals of neurology, 2016-11, Vol.80 (5), p.754-765

2016

Volltextzugriff (PDF)

Gene therapy for ALS: A review

Molecular therapy, 2021-12, Vol.29 (12), p.3345-3358

2021

Volltextzugriff (PDF)

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

Proceedings of the National Academy of Sciences - PNAS, 2008-04, Vol.105 (15), p.5868-5873

2008

Volltextzugriff (PDF)

In vivo SELEX for Identification of Brain-penetrating Aptamers

Molecular therapy. Nucleic acids, 2013-01, Vol.2 (1), p.e67-e67, Article e67

2013

Volltextzugriff (PDF)

Huntington’s disease phenotypes are improved via mTORC1 modulation by small molecule therapy

PloS one, 2022-08, Vol.17 (8), p.e0273710-e0273710

2022

Volltextzugriff (PDF)

Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching

Nature communications, 2023-05, Vol.14 (1), p.2628-2628, Article 2628

2023

Volltextzugriff (PDF)

1 – 25 von 358

Suche als RSS-Feed

Suchergebnisse filtern

Filter anzeigen

Nur zeigen

Geprüft (Peer-Review)
(347)
Mit Volltextzugriff
(314)

Publikationsform

Sprache

Englisch
(358)

Erscheinungsjahr

n.n

Thema

Science & Technology
(273)
Life Sciences & Biomedicine
(251)
Animals
(209)
Humans
(164)
Mice
(126)
Gene Therapy
(98)
Genetics & Heredity
(85)
Medicine, Research & Experimental
(78)
Research & Experimental Medicine
(78)
Gene Transfer Techniques
(77)
Biological And Medical Sciences
(72)
Genetic Vectors
(63)
Biotechnology & Applied Microbiology
(61)
Neurosciences & Neurology
(59)
Male
(58)
Neurosciences
(58)
Gene Expression
(53)
Adenoviridae - Genetics
(47)
Biochemistry & Molecular Biology
(47)
Disease Models, Animal
(46)

15 weitere anzeigen...

Weniger anzeigen...

Neu hinzugefügt

... seit 90 Tagen
(3)

Menü

Weitere Dienste

Einstellungen

Current prospects for RNA interference-based therapies

Reinstating aberrant mTORC1 activity in Huntington's disease mice improves disease phenotypes

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

Transcriptome-wide discovery of microRNA binding sites in human brain

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

The Bifunctional microRNA miR-9/miR-9 Regulates REST and CoREST and Is Downregulated in Huntington's Disease

Striatal neurons directly converted from Huntington's disease patient fibroblasts recapitulate age-associated disease phenotypes

DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencing

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion

Transvascular delivery of small interfering RNA to the central nervous system

Standard screening methods underreport AAV-mediated transduction and gene editing

Viral Vectors for Gene Transfer

PIAS1 Regulates Mutant Huntingtin Accumulation and Huntington’s Disease-Associated Phenotypes In Vivo

Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy

Structure and activity of putative intronic miRNA promoters

Safety and Efficacy of Ex Vivo Donor Lung Adenoviral IL-10 Gene Therapy in a Large Animal Lung Transplant Survival Model

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

RNAi prevents and reverses phenotypes induced by mutant human ataxin-1

Gene therapy for ALS: A review

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

In vivo SELEX for Identification of Brain-penetrating Aptamers

Huntington’s disease phenotypes are improved via mTORC1 modulation by small molecule therapy

Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching

Suchergebnisse filtern