Activating frataxin expression by repeat-targeted nucleic acids

Importin-β facilitates nuclear import of human GW proteins and balances cytoplasmic gene silencing protein levels

(2015) Daniel Schraivogel et al.   NUCLEIC ACIDS RESEARCH

RNAa in action: From the exception to the norm

(2015) Dan Guo et al.   RNA Biology

Long Non-Coding RNA Targeting and Transcriptional De-Repression

(2015) Marc S. Weinberg et al.   Nucleic Acid Therapeutics

Epigenetic therapy for Friedreich ataxia

(2014) Elisabetta Soragni et al.   ANNALS OF NEUROLOGY

Mechanisms of transcriptional dysregulation in repeat expansion disorders

(2014) Matthias Groh et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs

(2014) Jiaxin Hu et al.   BIOCHEMISTRY

Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's ataxia

(2014) Sunil Sahdeo et al.   HUMAN MOLECULAR GENETICS

Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich's ataxia: an exploratory, open-label, dose-escalation study

(2014) Vincenzo Libri et al.   LANCET

R-loops induce repressive chromatin marks over mammalian gene terminators

(2014) Konstantina Skourti-Stathaki et al.   NATURE

Oligonucleotide-based strategies to combat polyglutamine diseases

(2014) A. Fiszer et al.   NUCLEIC ACIDS RESEARCH

Import routes and nuclear functions of Argonaute and other small RNA-silencing proteins

(2014) Daniel Schraivogel et al.   TRENDS IN BIOCHEMICAL SCIENCES

RNAi Factors Are Present and Active in Human Cell Nuclei

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

R-loops Associated with Triplet Repeat Expansions Promote Gene Silencing in Friedreich Ataxia and Fragile X Syndrome

(2014) Matthias Groh et al.   PLoS Genetics

Transcription-Associated R-Loop Formation across the Human FMR1 CGG-Repeat Region

(2014) Erick W. Loomis et al.   PLoS Genetics

Epigenetic-based therapies for Friedreich ataxia

(2014) Chiranjeevi Sandi et al.   Frontiers in Genetics

Therapeutic strategies in Friedreich's Ataxia

(2013) Timothy E. Richardson et al.   BRAIN RESEARCH

Heterochromatinization induced by GAA-repeat hyperexpansion in Friedreich's ataxia can be reduced upon HDAC inhibition by vitamin B3

(2013) P. K. Chan et al.   HUMAN MOLECULAR GENETICS

Increasing frataxin gene expression with histone deacetylase inhibitors as a therapeutic approach for Friedreich's ataxia

(2013) Joel M. Gottesfeld et al.   JOURNAL OF NEUROCHEMISTRY

Clinical Neurogenetics

(2013) Abigail Collins   NEUROLOGIC CLINICS

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Promoter RNA links transcriptional regulation of inflammatory pathway genes

(2013) Masayuki Matsui et al.   NUCLEIC ACIDS RESEARCH

Chromatin changes in the development and pathology of the Fragile X-associated disorders and Friedreich ataxia

(2012) Daman Kumari et al.   Biochimica et Biophysica Acta-Gene Regulatory Mechanisms

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Friedreich's ataxia: Past, present and future

(2011) Daniele Marmolino   BRAIN RESEARCH REVIEWS

Locked vs. unlocked nucleic acids (LNA vs. UNA): contrasting structures work towards common therapeutic goals

(2011) Meghan A. Campbell et al.   CHEMICAL SOCIETY REVIEWS

Oligonucleotide therapeutic approaches for Huntington disease

(2011) Dinah W.Y. Sah et al.   JOURNAL OF CLINICAL INVESTIGATION

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

Prolonged treatment with pimelic o-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model

(2011) Chiranjeevi Sandi et al.   NEUROBIOLOGY OF DISEASE

Expanding the action of duplex RNAs into the nucleus: redirecting alternative splicing

(2011) Jing Liu et al.   NUCLEIC ACIDS RESEARCH

Long intronic GAA repeats causing Friedreich ataxia impede transcription elongation

(2010) Tanel Punga et al.   EMBO Molecular Medicine

Involvement of argonaute proteins in gene silencing and activation by RNAs complementary to a non-coding transcript at the progesterone receptor promoter

(2010) Yongjun Chu et al.   NUCLEIC ACIDS RESEARCH

Cerebrospinal fluid biomarkers in Guillain-Barré syndrome – Where do we stand?

(2009) Johannes Brettschneider et al.   JOURNAL OF NEUROLOGY

Control of alternative splicing through siRNA-mediated transcriptional gene silencing

(2009) Mariano Alló et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Structure of an argonaute silencing complex with a seed-containing guide DNA and target RNA duplex

(2008) Yanli Wang et al.   NATURE