Thierry VandenDriessche

Vrije Universiteit Brussel — Belgium

Biochemistry, Genetics and Molecular Biology · Life Sciences

55h-index11.0kcitations269works5.32yr avg
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Research Topics

Virus-based gene therapy research(143), CRISPR and Genetic Engineering(93), RNA Interference and Gene Delivery(50), CAR-T cell therapy research(47), Hemophilia Treatment and Research(34)

Publications269 total

Matchmaking gene therapy with mimetic antibodies
Blood·2025Open Access
Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional <i>Cis</i> -Regulatory Elements
Human Gene Therapy·2025· 2 cited
CRISPR and gene editing technologies for bleeding disorders
Therapeutic Advances in Hematology·2025Open Access
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease
Molecular Therapy — Methods & Clinical Development·2025· 5 citedOpen Access
Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors
Molecular Therapy — Methods & Clinical Development·2024· 9 citedOpen Access
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
Molecular Metabolism·2024· 16 citedOpen Access
Hemophilia Gene Therapy: The End of the Beginning?
Human Gene Therapy·2023· 32 cited
First hemophilia B gene therapy approved: More than two decades in the making
Molecular Therapy·2022· 30 citedOpen Access
Hemophilia “A” gene therapy: Lost in translation
Molecular Therapy·2022· 2 citedOpen Access
First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy
Molecular Therapy·2022· 29 citedOpen Access
Should patients with haemophilia receive gene therapy?
The Lancet Haematology·2022· 2 cited
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
Science Advances·2022· 83 citedOpen Access
Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy
Molecular Therapy·2022· 70 citedOpen Access
The RNA World: The Best of Times, the Worst of Times
Human Gene Therapy·2021
Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes
Molecular Therapy·2021· 16 citedOpen Access
Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element
Human Gene Therapy·2021· 10 cited
Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua
Blood·2021· 13 citedOpen Access
Liver-directed lentiviral gene therapy in a dog model of hemophilia B
UNC Libraries·2020Open Access
Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?
UNC Libraries·2020Open Access
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk
UNC Libraries·2020Open Access
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Frequent Co-authors

Marinee Chuah(46), Désiré Collen(7), Janka Mátrai(6), Abel Acosta‐Sanchez(5), Ermira Samara-Kuko(5), Lieven Thorrez(5), Nisha Nair(5), Peter Carmeliet(4), Ling Ma(4), Warut Tulalamba(4), Luigi Naldini(4), Melvin Y. Rincón(4), Jaitip Tipanee(4), Lieve Moons(3), D Collen(3), Zoltán Ivics(3), Zsuzsanna Izsvák(3), Inge Petrus(3), Veerle Gillijns(3), Veerle Vanslembrouck(3)