Cargando…

Ex vivo Improvement of a von Willebrand Disease Type 2A Phenotype Using an Allele-Specific Small-Interfering RNA

Von Willebrand disease (VWD) is the most common inherited bleeding disorder and is mainly caused by dominant-negative mutations in the multimeric protein von Willebrand factor (VWF). These mutations may either result in quantitative or qualitative defects in VWF. VWF is an endothelial protein that i...

Descripción completa

Detalles Bibliográficos
Autores principales:	de Jong, Annika, Dirven, Richard J., Boender, Johan, Atiq, Ferdows, Anvar, Seyed Yahya, Leebeek, Frank W. G., van Vlijmen, Bart J. M., Eikenboom, Jeroen
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Georg Thieme Verlag KG 2020
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7649061/ https://www.ncbi.nlm.nih.gov/pubmed/32803740 http://dx.doi.org/10.1055/s-0040-1715442

Ejemplares similares

Small interfering RNA–mediated allele-selective silencing of von Willebrand factor in vitro and in vivo
por: Jongejan, Yvonne K., et al.
Publicado: (2023)

Von Willebrand Factor Multimer Densitometric Analysis: Validation of the Clinical Accuracy and Clinical Implications in Von Willebrand Disease
por: Boender, Johan, et al.
Publicado: (2021)

How I manage severe von Willebrand disease
por: Leebeek, Frank W. G., et al.
Publicado: (2019)

Importance of Genotyping in von Willebrand Disease to Elucidate Pathogenic Mechanisms and Variability in Phenotype
por: Atiq, Ferdows, et al.
Publicado: (2022)

Variability of von Willebrand factor‐related parameters in endothelial colony forming cells
por: de Jong, Annika, et al.
Publicado: (2019)

Comorbidities associated with higher von Willebrand factor (VWF) levels may explain the age‐related increase of VWF in von Willebrand disease
por: Atiq, Ferdows, et al.
Publicado: (2018)

Sports participation and physical activity in patients with von Willebrand disease
por: Atiq, Ferdows, et al.
Publicado: (2018)

Desmopressin testing in von Willebrand disease: Lowering the burden
por: Heijdra, Jessica M., et al.
Publicado: (2022)

Quantitative 3D microscopy highlights altered von Willebrand factor α‐granule storage in patients with von Willebrand disease with distinct pathogenic mechanisms
por: Swinkels, Maurice, et al.
Publicado: (2021)

ADAMTS‐13 and bleeding phenotype in von Willebrand disease
por: Boender, Johan, et al.
Publicado: (2020)

Criteria for low von Willebrand factor diagnosis and risk score to predict future bleeding
por: Atiq, Ferdows, et al.
Publicado: (2021)

BMI is an important determinant of VWF and FVIII levels and bleeding phenotype in patients with von Willebrand disease
por: Atiq, Ferdows, et al.
Publicado: (2019)

Desmopressin response depends on the presence and type of genetic variants in patients with type 1 and type 2 von Willebrand disease
por: Atiq, Ferdows, et al.
Publicado: (2022)

Social participation is reduced in type 3 Von Willebrand disease patients and in patients with a severe bleeding phenotype
por: Kempers, Eva K., et al.
Publicado: (2021)

Platelet degranulation and bleeding phenotype in a large cohort of Von Willebrand disease patients
por: Swinkels, Maurice, et al.
Publicado: (2022)

Von Willebrand Disease: From In Vivo to In Vitro Disease Models
por: de Boer, Suzan, et al.
Publicado: (2019)

Von Willebrand disease type 2M: Correlation between genotype and phenotype
por: Maas, Dominique P. M. S. M., et al.
Publicado: (2021)

Von Willebrand factor and ADAMTS13 activity in relation to risk of dementia: a population-based study
por: Wolters, Frank J., et al.
Publicado: (2018)

von Willebrand factor levels in the diagnosis of von Willebrand disease: a systematic review and meta-analysis
por: Kalot, Mohamad A., et al.
Publicado: (2021)

Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results
por: Leebeek, Frank W. G., et al.
Publicado: (2022)

New Developments in Diagnosis and Management of Acquired Hemophilia and Acquired von Willebrand Syndrome
por: Leebeek, Frank W. G.
Publicado: (2021)

Current and Emerging Options for the Management of Inherited von Willebrand Disease
por: Heijdra, Jessica M., et al.
Publicado: (2017)

Interaction of von Willebrand factor with blood cells in flow models: a systematic review
por: Arisz, Ryanne A., et al.
Publicado: (2022)

Von Willebrand factor propeptide and pathophysiological mechanisms in European and Iranian patients with type 3 von Willebrand disease enrolled in the 3WINTERS‐IPS study
por: Pagliari, Maria Teresa, et al.
Publicado: (2022)

Effect of Genetic Variation in STXBP5 and STX2 on von Willebrand Factor and Bleeding Phenotype in Type 1 von Willebrand Disease Patients
por: van Loon, Janine E., et al.
Publicado: (2012)

Long-term impact of joint bleeds in von Willebrand disease: a nested case-control study
por: van Galen, Karin P.M., et al.
Publicado: (2017)

Hemophilia and von Willebrand disease: Factor VIII and von Willebrand factor
por: Sharma, Prashant
Publicado: (2019)

Diagnostic Value of Measuring Platelet Von Willebrand Factor in Von Willebrand Disease
por: Casonato, Alessandra, et al.
Publicado: (2016)

Classification of von Willebrand disease in the context of modern contemporary von Willebrand factor testing methodologies
por: Favaloro, Emmanuel J.
Publicado: (2020)

Of von Willebrand factor and platelets
por: Bryckaert, Marijke, et al.
Publicado: (2014)

One piece of the puzzle: Population pharmacokinetics of FVIII during perioperative Haemate P(®)/Humate P(®) treatment in von Willebrand disease patients
por: de Jager, Nico C. B., et al.
Publicado: (2019)

On the Versatility of von Willebrand Factor
por: Rauch, Antoine, et al.
Publicado: (2013)

Changes of von Willebrand Factor during Pregnancy in Women with and without von Willebrand Disease
por: Castaman, Giancarlo
Publicado: (2013)

Genetic Variation in the von Willebrand Factor Gene in Swedish von Willebrand Disease Patients
por: Manderstedt, Eric, et al.
Publicado: (2018)

Is pharmacokinetic-guided dosing of desmopressin and von Willebrand factor-containing concentrates in individuals with von Willebrand disease or low von Willebrand factor reliable and feasible? A protocol for a multicentre, non-randomised, open label cohort trial, the OPTI-CLOT: to WiN study
por: Heijdra, Jessica M, et al.
Publicado: (2022)

Von Willebrand Disease: An Overview
por: Bharati, K. Pavani, et al.
Publicado: (2011)

Porcine and Canine von Willebrand Factor and von Willebrand Disease: Hemostasis, Thrombosis, and Atherosclerosis Studies
por: Nichols, Timothy C., et al.
Publicado: (2010)

Improving diagnosis of von Willebrand disease: Reference ranges for von Willebrand factor multimer distribution
por: Vangenechten, Inge, et al.
Publicado: (2020)

Differences in von Willebrand factor function in type 2A von Willebrand disease and left ventricular assist device‐induced acquired von Willebrand syndrome
por: Deconinck, Shannen, et al.
Publicado: (2018)

Performance Related Factors Are the Main Determinants of the von Willebrand Factor Response to Exhaustive Physical Exercise
por: van Loon, Janine E., et al.
Publicado: (2014)

Cannot write session to /tmp/vufind_sessions/sess_9rfu8dor4dn2eev6v6kv9orjh1