RNAi: a potential new class of therapeutic for human genetic disease

MicroRNAs in Sjögren's syndrome as a prototypic autoimmune disease

(2010) Ilias Alevizos et al.   AUTOIMMUNITY REVIEWS

AKT1 and AKT2 maintain hematopoietic stem cell function by regulating reactive oxygen species

(2010) M. M. Juntilla et al.   BLOOD

RNAi Screening for the Discovery of Novel Modulators of Human Disease

(2010) Attila A. Seyhan et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Inhibitory RNA in epilepsy: Research tools and therapeutic perspectives

(2010) Detlev Boison   EPILEPSIA

Gene therapy strategies for hemophilia: benefits versus risks

(2010) Inge Petrus et al.   JOURNAL OF GENE MEDICINE

Cancer: The blind spot of p53

(2010) Anton Berns   NATURE

Stage-specific sensitivity to p53 restoration during lung cancer progression

(2010) David M. Feldser et al.   NATURE

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

(2010) Mark E. Davis et al.   NATURE

MicroRNAs as biomarkers in rheumatic diseases

(2010) Ilias Alevizos et al.   Nature Reviews Rheumatology

171st ENMC International Workshop: Standards of care and management of facioscapulohumeral muscular dystrophy

(2010) Rabi Tawil et al.   NEUROMUSCULAR DISORDERS

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

In vivo RNAi: Today and Tomorrow

(2010) N. Perrimon et al.   Cold Spring Harbor Perspectives in Biology

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

Progress and Challenges in RNA Interference Therapy for Huntington Disease

(2009) Scott Q. Harper   ARCHIVES OF NEUROLOGY

RNAi as a new therapeutic strategy against HCV

(2009) Saba Khaliq et al.   BIOTECHNOLOGY ADVANCES

Importin 8 Is a Gene Silencing Factor that Targets Argonaute Proteins to Distinct mRNAs

(2009) Lasse Weinmann et al.   CELL

MicroRNAs: Target Recognition and Regulatory Functions

(2009) David P. Bartel   CELL

MicroRNAs in development and disease

(2009) AE Erson et al.   CLINICAL GENETICS

Harnessing RNA interference to develop neonatal therapies: From Nobel Prize winning discovery to proof of concept clinical trials

(2009) John P. DeVincenzo   EARLY HUMAN DEVELOPMENT

RNA interference screening for the discovery of oncology targets

(2009) Kim Quon et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

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

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

Allele-specific RNAi Mitigates Phenotypic Progression in a Transgenic Model of Alzheimer's Disease

(2009) Edgardo Rodríguez-Lebrón et al.   MOLECULAR THERAPY

First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder

(2009) Sancy A Leachman et al.   MOLECULAR THERAPY

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

(2009) Steven J Gray et al.   MOLECULAR THERAPY

The promises and pitfalls of RNA-interference-based therapeutics

(2009) Daniela Castanotto et al.   NATURE

Suppression of hippocampal TRPM7 protein prevents delayed neuronal death in brain ischemia

(2009) Hong-Shuo Sun et al.   NATURE NEUROSCIENCE

Small silencing RNAs: an expanding universe

(2009) Megha Ghildiyal et al.   NATURE REVIEWS GENETICS

Neuropeptide Y overexpression using recombinant adenoassociated viral vectors

(2009) Francesco Noé et al.   Neurotherapeutics

Design of RNAi Hairpins for Mutation-Specific Silencing of Ataxin-7 and Correction of a SCA7 Phenotype

(2009) Janine Scholefield et al.   PLoS One

RNAi mechanisms and applications

(2008) Daniel H. Kim et al.   BIOTECHNIQUES

T Cell-Specific siRNA Delivery Suppresses HIV-1 Infection in Humanized Mice

(2008) Priti Kumar et al.   CELL

Gene therapy in epilepsy

(2008) Véronique Riban et al.   EPILEPSIA

Therapeutic siRNAs for dominant genetic skin disorders including pachyonychia congenita

(2008) Sancy A. Leachman et al.   JOURNAL OF DERMATOLOGICAL SCIENCE

An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models

(2008) Maria A Graziewicz et al.   MOLECULAR THERAPY

Novel Dual Inhibitory Function Aptamer–siRNA Delivery System for HIV-1 Therapy

(2008) Jiehua Zhou et al.   MOLECULAR THERAPY

AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

(2008) Nicholas R Franich et al.   MOLECULAR THERAPY

Central Delivery of Dicer-substrate siRNA: A Direct Application for Pain Research

(2008) Louis Doré-Savard et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Fluorescence correlation spectroscopy and fluorescence cross-correlation spectroscopy reveal the cytoplasmic origination of loaded nuclear RISC in vivo in human cells

(2008) Thomas Ohrt et al.   NUCLEIC ACIDS RESEARCH

Chemical Modification of siRNAs for In Vivo Use

(2008) Mark A. Behlke   OLIGONUCLEOTIDES

Enhancement of Allele Discrimination by Introduction of Nucleotide Mismatches into siRNA in Allele-Specific Gene Silencing by RNAi

(2008) Yusuke Ohnishi et al.   PLoS One

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

Single treatment with RNAi against prion protein rescues early neuronal dysfunction and prolongs survival in mice with prion disease

(2008) M. D. White et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Discovery, biology and therapeutic potential of RNA interference, microRNA and antagomirs

(2007) Ming Yang et al.   PHARMACOLOGY & THERAPEUTICS