Thierry Vandendriessche
Brussels, BRU, Belgium
Last Updated: 09/26/2026
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Found 75 publications
Increased gene therapy efficacy through the use of extended half-life clotting factors.
Journal: Blood advances
Published: October 24, 2025
Increased Gene Therapy Efficacy Through the Use of Extended Half-Life Clotting Factors.
Journal: Blood advances
Published: October 24, 2025
Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis-Regulatory Elements.
Journal: Human gene therapy
Published: July 18, 2025
CRISPR and gene editing technologies for bleeding disorders.
Journal: Therapeutic advances in hematology
Published: April 24, 2025
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.
Journal: Molecular therapy. Methods & clinical development
Published: January 09, 2025
Hemophilia Gene Therapy: The End of the Beginning?
Journal: Human gene therapy
Published: September 06, 2023
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.
Journal: Molecular metabolism
Published: July 31, 2023
Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors.
Journal: Molecular therapy. Methods & clinical development
Published: July 20, 2023
First hemophilia B gene therapy approved: More than two decades in the making.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: December 07, 2022
Hemophilia "A" gene therapy: Lost in translation.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: November 10, 2022
Should patients with haemophilia receive gene therapy?
Journal: The Lancet. Haematology
Published: September 29, 2022
Last Updated: 09/26/2026