Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy

Systemic RNAi Delivery to the Muscles of ROSA26 Mice Reduces lacZ Expression

(2014) Jessica Wei et al.   PLoS One

Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy

(2014) M. K. Childers et al.   Science Translational Medicine

RNAi Factors Are Present and Active in Human Cell Nuclei

(2014) Keith T. Gagnon et al.   Cell Reports

Reducing Levels of Toxic RNA with Small Molecules

(2013) Leslie A. Coonrod et al.   ACS Chemical Biology

Splicing biomarkers of disease severity in myotonic dystrophy

(2013) Masayuki Nakamori et al.   ANNALS OF NEUROLOGY

Compound loss of muscleblind-like function in myotonic dystrophy

(2013) Kuang-Yung Lee et al.   EMBO Molecular Medicine

Evaluating the effects of CELF1 deficiency in a mouse model of RNA toxicity

(2013) Yun Kyoung Kim et al.   HUMAN MOLECULAR GENETICS

High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines

(2013) Ami Ketley et al.   HUMAN MOLECULAR GENETICS

Recombinant AAV as a Platform for Translating the Therapeutic Potential of RNA Interference

(2013) Florie Borel et al.   MOLECULAR THERAPY

RNA interference in the nucleus: roles for small RNAs in transcription, epigenetics and beyond

(2013) Stephane E. Castel et al.   NATURE REVIEWS GENETICS

Intracellular Localization and Routing of miRNA and RNAi Pathway Components

(2012) Thomas Ohrt et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Design of a Bioactive Small Molecule That Targets the Myotonic Dystrophy Type 1 RNA via an RNA Motif–Ligand Database and Chemical Similarity Searching

(2012) Raman Parkesh et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

The myotonic dystrophies: molecular, clinical, and therapeutic challenges

(2012) Bjarne Udd et al.   LANCET NEUROLOGY

RNA Interference Targeting CUG Repeats in a Mouse Model of Myotonic Dystrophy

(2012) Krzysztof Sobczak et al.   MOLECULAR THERAPY

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

(2012) Thurman M. Wheeler et al.   NATURE

RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1

(2012) J. E. Lee et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

AAV6-mediated Systemic shRNA Delivery Reverses Disease in a Mouse Model of Facioscapulohumeral Muscular Dystrophy

(2011) Sergia Bortolanza et al.   MOLECULAR THERAPY

RNA Interference Improves Myopathic Phenotypes in Mice Over-expressing FSHD Region Gene 1 (FRG1)

(2011) Lindsay M Wallace et al.   MOLECULAR THERAPY

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophy

(2011) Charlotte Fugier et al.   NATURE MEDICINE

Current prospects for RNA interference-based therapies

(2011) Beverly L. Davidson et al.   NATURE REVIEWS GENETICS

Depletion of key protein components of the RISC pathway impairs pre-ribosomal RNA processing

(2011) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

Myotonic dystrophy mouse models: towards rational therapy development

(2011) Mário Gomes-Pereira et al.   TRENDS IN MOLECULAR MEDICINE

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

(2010) Dirk Grimm et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

The promises and pitfalls of RNA-interference-based therapeutics

(2009) Daniela Castanotto et al.   NATURE

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

(2009) S. A. M. Mulders et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

(2009) T. M. Wheeler et al.   SCIENCE

Biodistribution and Safety Profile of Recombinant Adeno-Associated Virus Serotype 6 Vectors following Intravenous Delivery

(2008) D. Stone et al.   JOURNAL OF VIROLOGY

Expression of shRNA From a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic

(2008) Jeffery C Giering et al.   MOLECULAR THERAPY

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

(2008) Ryan L Boudreau et al.   MOLECULAR THERAPY

Expanded CTG repeats within the DMPK 3' UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy

(2008) J. P. Orengo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs

(2008) R. L. Boudreau et al.   RNA