Correction of a dominant-negative von Willebrand factor multimerization defect by small interfering RNA-mediated allele-specific inhibition of mutant von Willebrand factor

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

(2017) K. John Pasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Von Willebrand disease mutation spectrum and associated mutation mechanisms

(2017) Annika de Jong et al.   THROMBOSIS RESEARCH

Characterization of aberrant splicing of von Willebrand factor in von Willebrand disease: an underrecognized mechanism

(2016) L. Hawke et al.   BLOOD

Von Willebrand’s Disease

(2016) Frank W.G. Leebeek et al.   NEW ENGLAND JOURNAL OF MEDICINE

Treatment of von Willebrand Disease

(2016) Leonardo Pasalic et al.   SEMINARS IN THROMBOSIS AND HEMOSTASIS

Allele-specific silencing of EEC p63 mutant R304W restores p63 transcriptional activity

(2016) F Novelli et al.   Cell Death & Disease

Von Willebrand factor (Vonvendi®): the first recombinant product licensed for the treatment of von Willebrand disease

(2016) Massimo Franchini et al.   Expert Review of Hematology

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

(2016) Jana Miniarikova et al.   Molecular Therapy-Nucleic Acids

Unexpectedly High Prevalence of Acquired von Willebrand Syndrome in Patients with Severe Aortic Stenosis as Evaluated with a Novel Large Multimer Index

(2015) Toshihiro Tamura et al.   Journal of Atherosclerosis and Thrombosis

Angiogenic characteristics of blood outgrowth endothelial cells from patients with von Willebrand disease

(2015) D. J. Groeneveld et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

A global reference for human genetic variation

(2015) Richard A. Gibbs et al.   NATURE

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Delivery of Therapeutic siRNA to the Lung Endothelium via Novel Lipoplex Formulation DACC

(2014) V Fehring et al.   MOLECULAR THERAPY

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

(2014) Niels H. Skotte et al.   PLoS One

Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR–Cas platform

(2014) K. Yoshimi et al.   Nature Communications

von Willebrand disease: advances in pathogenetic understanding, diagnosis, and therapy

(2013) D. Lillicrap   BLOOD

Analysis of the storage and secretion of von Willebrand factor in blood outgrowth endothelial cells derived from patients with von Willebrand disease

(2013) J.-W. Wang et al.   BLOOD

Cellular and molecular basis of von Willebrand disease: studies on blood outgrowth endothelial cells

(2013) R. D. Starke et al.   BLOOD

Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients

(2012) G. CASTAMAN et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Establishment of outgrowth endothelial cells from peripheral blood

(2012) Javier Martin-Ramirez et al.   Nature Protocols

The dominant-negative von Willebrand factor gene deletion p.P1127_C1948delinsR: molecular mechanism and modulation

(2010) C. Casari et al.   BLOOD

Endothelial von Willebrand factor regulates angiogenesis

(2010) R. D. Starke et al.   BLOOD

The genetic basis of von Willebrand disease

(2010) Anne C. Goodeve   BLOOD REVIEWS

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY