Yuji Kashiwakura

Tochigi, Japan

Last Updated: 09/26/2026

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Found 36 publications

High Prevalence of Preexisting Neutralizing Antibodies Against Adeno-Associated Virus Capsids in Mongolian People with Hemophilia.

High Prevalence of Preexisting Neutralizing Antibodies Against Adeno-Associated Virus Capsids in Mongolian People with Hemophilia.

Journal: Human gene therapy

Published: July 27, 2026

Nonviral delivery of a base editor enables personalized correction of hemophilia B nonsense variants in a mouse model.

Nonviral delivery of a base editor enables personalized correction of hemophilia B nonsense variants in a mouse model.

Journal: Journal of thrombosis and haemostasis : JTH

Published: November 10, 2025

Engineering a compact high-fidelity Staphylococcus aureus Cas9 variant with broader targeting range and mechanistic insights into its activation.

Engineering a compact high-fidelity Staphylococcus aureus Cas9 variant with broader targeting range and mechanistic insights into its activation.

Journal: Nature communications

Published: September 14, 2025

Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors.

Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors.

Journal: Molecular therapy. Advances

Published: September 03, 2025

Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy.

Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy.

Journal: Blood

Published: January 17, 2025

The factor (F)VIII K1693N mutation (FVIII-Nara) in a patient with moderate hemophilia A confers resistance to thrombin-catalyzed cleavage at Arg1689 involving P4' position.

The factor (F)VIII K1693N mutation (FVIII-Nara) in a patient with moderate hemophilia A confers resistance to thrombin-catalyzed cleavage at Arg1689 involving P4' position.

Journal: Journal of thrombosis and haemostasis : JTH

Published: December 10, 2024

Therapeutic base editing to generate a gain-of-function F9 variant for hemophilia B.

Therapeutic base editing to generate a gain-of-function F9 variant for hemophilia B.

Journal: Blood

Published: November 30, 2024

Cure of Congenital Purpura Fulminans via Expression of Engineered Protein C Through Neonatal Genome Editing in Mice.

Cure of Congenital Purpura Fulminans via Expression of Engineered Protein C Through Neonatal Genome Editing in Mice.

Journal: Arteriosclerosis, thrombosis, and vascular biology

Published: November 07, 2024

Glycosylation of recombinant adeno-associated virus serotype 6.

Glycosylation of recombinant adeno-associated virus serotype 6.

Journal: Molecular therapy. Methods & clinical development

Published: February 08, 2024

The combination of Asp519Val/Glu665Val and Lys1813Ala mutations in FVIII markedly increases coagulation potential.

The combination of Asp519Val/Glu665Val and Lys1813Ala mutations in FVIII markedly increases coagulation potential.

Journal: Blood advances

Published: December 12, 2023

Current status and future perspective of treatment for hemophilia

Current status and future perspective of treatment for hemophilia

Journal: [Rinsho ketsueki] The Japanese journal of clinical hematology

Published: June 04, 2023

Genome editing of patient-derived iPSCs identifies a deep intronic variant causing aberrant splicing in hemophilia A.

Genome editing of patient-derived iPSCs identifies a deep intronic variant causing aberrant splicing in hemophilia A.

Journal: Blood advances

Published: May 30, 2023

Showing 1-12 of 36

Last Updated: 09/26/2026