Cargando…

Blockade of the costimulatory CD28‐B7 family signal axis enables repeated application of AAV8 gene vectors

Adeno‐associated virus serotype 8 (AAV8) gene therapy has shown efficacy in several clinical trials and is considered a highly promising technology to treat monogenic diseases such as hemophilia A and B. However, a major drawback of AAV8 gene therapy is that it can be applied only once because anti‐...

Descripción completa

Detalles Bibliográficos
Autores principales:	Frentsch, Marco, Japp, Alberto Sada, Dingeldey, Manuela, Matzmohr, Nadine, Thiel, Andreas, Scheiflinger, Friedrich, Reipert, Birgit M., de la Rosa, Maurus
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	HAEMOSTASIS
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7318590/ https://www.ncbi.nlm.nih.gov/pubmed/32011092 http://dx.doi.org/10.1111/jth.14757

Ejemplares similares

Temperature‐dependent irreversible conformational change of recombinant ADAMTS13 upon metal ion chelation
por: Rottensteiner, Hanspeter, et al.
Publicado: (2019)

High-dimensional single cell mass cytometry analysis of the murine hematopoietic system reveals signatures induced by ageing and physiological pathogen challenges
por: Nikolaou, Christos, et al.
Publicado: (2021)

Correction to: High-dimensional single cell mass cytometry analysis of the murine hematopoietic system reveals signatures induced by ageing and physiological pathogen challenges
por: Nikolaou, Christos, et al.
Publicado: (2021)

Long‐term follow‐up of patients treated with caplacizumab and safety and efficacy of repeat caplacizumab use: Post‐HERCULES study
por: Scully, Marie, et al.
Publicado: (2022)

Pharmacokinetics, immunogenicity, safety, and preliminary efficacy of subcutaneous turoctocog alfa pegol in previously treated patients with severe hemophilia A (alleviate 1)
por: Klamroth, Robert, et al.
Publicado: (2019)

Long‐term safety and efficacy of rIX‐FP prophylaxis with extended dosing intervals up to 21 days in adults/adolescents with hemophilia B
por: Mancuso, Maria Elisa, et al.
Publicado: (2020)

Influence of blood group, von Willebrand factor levels, and age on factor VIII levels in non‐severe haemophilia A
por: Rejtő, Judit, et al.
Publicado: (2020)

Evaluation of oral factor Xa inhibitor‐associated extracranial bleeding reversal with andexanet alfa
por: Nederpelt, Charlie J., et al.
Publicado: (2020)

The experiences and attitudes of hemophilia carriers around pregnancy: A qualitative systematic review
por: Punt, Marieke C., et al.
Publicado: (2020)

A novel assay for studying the involvement of blood cells in whole blood thrombin generation
por: Wan, Jun, et al.
Publicado: (2020)

Coagulopathy signature precedes and predicts severity of end‐organ heat stroke pathology in a mouse model
por: Proctor, Elizabeth A., et al.
Publicado: (2020)

Management of von Willebrand disease with a factor VIII‐poor von Willebrand factor concentrate: Results from a prospective observational post‐marketing study
por: Goudemand, Jenny, et al.
Publicado: (2020)

Performance of factor IX extended half‐life product measurements in external quality control assessment programs
por: Nederlof, Angelique, et al.
Publicado: (2020)

Efficacy of pro‐ and anticoagulant strategies in plasma of patients undergoing hepatobiliary surgery
por: Bos, Sarah, et al.
Publicado: (2020)

The direct oral anticoagulants rivaroxaban and dabigatran do not inhibit orthotopic growth and metastasis of human breast cancer in mice
por: Buijs, Jeroen T., et al.
Publicado: (2019)

Intrinsic differences between FVIIIa mimetic bispecific antibodies and FVIII prevent assignment of FVIII‐equivalence
por: Leksa, Nina C., et al.
Publicado: (2019)

Thromboelastography‐guided therapy improves patient blood management and certain clinical outcomes in elective cardiac and liver surgery and emergency resuscitation: A systematic review and analysis
por: Dias, João D., et al.
Publicado: (2019)

Thrombin‐generating potential, plasma clot formation, and clot lysis are impaired in patients with bleeding of unknown cause
por: Hofer, Stefanie, et al.
Publicado: (2019)

Effect of multidose drug dispensing on the time in therapeutic range in patients using vitamin‐K antagonists: A randomized controlled trial
por: Mertens, Bram J., et al.
Publicado: (2019)

Validity and reliability of the Colorado Adult Joint Assessment Scale in adults with moderate‐severe hemophilia A
por: Funk, Sharon M., et al.
Publicado: (2019)

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)

Efficacy of fibrinogen concentrate in major abdominal surgery – A prospective, randomized, controlled study in cytoreductive surgery for pseudomyxoma peritonei
por: Roy, Ashok, et al.
Publicado: (2019)

Hydrogen‐deuterium exchange mass spectrometry highlights conformational changes induced by factor XI activation and binding of factor IX to factor XIa
por: Bar Barroeta, Awital, et al.
Publicado: (2019)

Drug levels and bleeding complications in atrial fibrillation patients treated with direct oral anticoagulants
por: Testa, Sophie, et al.
Publicado: (2019)

Fibrinogen αC‐regions are not directly involved in fibrin polymerization as evidenced by a “Double‐Detroit” recombinant fibrinogen mutant and knobs‐mimic peptides
por: Duval, Cédric, et al.
Publicado: (2020)

Fibrinogen concentrate for treatment of bleeding and surgical prophylaxis in congenital fibrinogen deficiency patients
por: Lissitchkov, Toshko, et al.
Publicado: (2020)

Whole blood thrombin generation profiles of patients with cirrhosis explored with a near patient assay
por: Wan, Jun, et al.
Publicado: (2020)

Global hemostatic status in patients with acute‐on‐chronic liver failure and septics without underlying liver disease
por: Lisman, Ton, et al.
Publicado: (2020)

A clinical prediction score for transient versus persistent childhood immune thrombocytopenia
por: Schmidt, David E., et al.
Publicado: (2020)

Mortality, life expectancy, and causes of death of persons with hemophilia in the Netherlands 2001–2018
por: Hassan, Shermarke, et al.
Publicado: (2020)

Prophylactic fresh frozen plasma and platelet transfusion have a prothrombotic effect in patients with liver disease
por: von Meijenfeldt, Fien A., 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)

Liver transplantation as a novel strategy for resolution of congenital afibrinogenemia in a pediatric patient
por: Corrales‐Medina, Fernando F., et al.
Publicado: (2020)

Pharmacodynamic monitoring of factor VIII replacement therapy in hemophilia A: Combining thrombin and plasmin generation
por: Valke, Lars L.F.G., et al.
Publicado: (2020)

The factor VIII treatment history of non‐severe hemophilia A
por: Abdi, Amal, et al.
Publicado: (2020)

Development of a novel fully functional coagulation factor VIII with reduced immunogenicity utilizing an in silico prediction and deimmunization approach
por: Winterling, Karina, et al.
Publicado: (2021)

Treatment‐related risk factors for inhibitor development in non‐severe hemophilia A after 50 cumulative exposure days: A case‐control study
por: Abdi, Amal, et al.
Publicado: (2021)

Structure‐function of anticoagulant TIX‐5, the inhibitor of factor Xa‐mediated FV activation
por: Maag, Anja, et al.
Publicado: (2021)

FVIII inhibitors display FV‐neutralizing activity in the prothrombin time assay
por: Arsiccio, Andrea, et al.
Publicado: (2021)

Thrombin generation potential in the presence of concizumab and rFVIIa, APCC, rFVIII, or rFIX: In vitro and ex vivo analyses
por: Kjalke, Marianne, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_9dsfvv7kfgofj8iqf8bq7uu7cu