Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes

In Vitro Uses of Human Pluripotent Stem Cell-Derived Cardiomyocytes

(2012) Elena Matsa et al.   Journal of Cardiovascular Translational Research

Pulmonary delivery of therapeutic siRNA

(2011) Jenny Ka-Wing Lam et al.   ADVANCED DRUG DELIVERY REVIEWS

Genetic Testing for Potentially Lethal, Highly Treatable Inherited Cardiomyopathies/Channelopathies in Clinical Practice

(2011) David J. Tester et al.   CIRCULATION

Drug evaluation in cardiomyocytes derived from human induced pluripotent stem cells carrying a long QT syndrome type 2 mutation

(2011) Elena Matsa et al.   EUROPEAN HEART JOURNAL

Allele-specific siRNA knockdown as a personalized treatment strategy for vascular Ehlers-Danlos syndrome in human fibroblasts

(2011) Gerd A. Müller et al.   FASEB JOURNAL

Genome Engineering With Zinc-Finger Nucleases

(2011) Dana Carroll   GENETICS

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

(2011) Sebahattin Cirak et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Faster generation of hiPSCs by coupling high-titer lentivirus and column-based positive selection

(2011) Emily Dick et al.   Nature Protocols

Trafficking Defect and Proteasomal Degradation Contribute to the Phenotype of a Novel KCNH2 Long QT Syndrome Mutation

(2011) Anton Mihic et al.   PLoS One

A Universal System for Highly Efficient Cardiac Differentiation of Human Induced Pluripotent Stem Cells That Eliminates Interline Variability

(2011) Paul W. Burridge et al.   PLoS One

Pluripotent stem cell models of cardiac disease and their implication for drug discovery and development

(2011) Richard P. Davis et al.   TRENDS IN MOLECULAR MEDICINE

RNAi-Based Treatment for Neovascular Age-Related Macular Degeneration by Sirna-027

(2010) Peter K. Kaiser et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Molecular genetics of long QT syndrome

(2010) Nilesh J. Bokil et al.   MOLECULAR GENETICS AND METABOLISM

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

(2010) Mark E. Davis et al.   NATURE

Tailor-made RNAi knockdown against triplet repeat disease-causing alleles

(2010) M. Takahashi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Two new protocols to enhance the production and isolation of human induced pluripotent stem cell lines

(2010) Emily Dick et al.   Stem Cell Research

Maintenance of pluripotency in human embryonic stem cells cultured on a synthetic substrate in conditioned medium

(2009) Magdalena M. Mahlstedt et al.   BIOTECHNOLOGY AND BIOENGINEERING

HERG-F463L POTASSIUM CHANNELS LINKED TO LONG QT SYNDROME REDUCEIKrCURRENT BY A TRAFFICKING-DEFICIENT MECHANISM

(2009) Hai-Tao Yang et al.   CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY

Profiling of mismatch discrimination in RNAi enabled rational design of allele-specific siRNAs

(2009) Huang Huang et al.   NUCLEIC ACIDS RESEARCH

The QT syndromes: long and short

(2008) Hiroshi Morita et al.   LANCET

Feeder-free culture of human embryonic stem cells in conditioned medium for efficient genetic modification

(2008) Stefan R Braam et al.   Nature Protocols

Design and Synthesis of N 4,N 9-Disubstituted Spermines for Non-viral siRNA Delivery – Structure-Activity Relationship Studies of siFection Efficiency Versus Toxicity

(2008) Moustafa K. Soltan et al.   PHARMACEUTICAL RESEARCH