Marinee Chuah

Vrije Universiteit Brussel — Belgium

Biochemistry, Genetics and Molecular Biology · Life Sciences

53h-index8.0kcitations220works5.32yr avg
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Research Topics

Virus-based gene therapy research(122), CRISPR and Genetic Engineering(83), RNA Interference and Gene Delivery(40), CAR-T cell therapy research(32), Hemophilia Treatment and Research(30)

Publications220 total

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
6463 A Case of Tumour-Induced Osteomalacia Which Remitted After Treatment of Metastatic Prostate Cancer
Journal of the Endocrine Society·2024Open 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
Hemophilia “A” gene therapy: Lost in translation
Molecular Therapy·2022· 2 citedOpen Access
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
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
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk
UNC Libraries·2020Open Access
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants
Nature Communications·2020· 247 citedOpen Access
Genetic and Epigenetic Modification of Rat Liver Progenitor Cells via HNF4α Transduction and 5’ Azacytidine Treatment: An Integrated miRNA and mRNA Expression Profile Analysis
Genes·2020· 3 citedOpen Access
Validation of miR-20a as a Tumor Suppressor Gene in Liver Carcinoma Using Hepatocyte-Specific Hyperactive piggyBac Transposons
Molecular Therapy — Nucleic Acids·2020· 12 citedOpen Access
Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys
Blood·2019· 2 cited
Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors
Gene Therapy·2019· 23 cited
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Frequent Co-authors

Thierry VandenDriessche(49), Désiré Collen(7), Janka Mátrai(6), Nisha Nair(6), Abel Acosta‐Sanchez(5), Ermira Samara-Kuko(5), Lieven Thorrez(5), Ling Ma(4), Warut Tulalamba(4), Luigi Naldini(4), Melvin Y. Rincón(4), Désiré Collen(4), Jaitip Tipanee(4), Zoltán Ivics(3), Zsuzsanna Izsvák(3), Inge Petrus(3), Veerle Gillijns(3), Veerle Vanslembrouck(3), Alessio Cantore(3), David Lillicrap(3)