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M Chuah

Katholieke Universiteit Leuven

20

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111

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Next-generation muscle-directed gene therapy by in silico vector design

2019 Nature Communications
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CRISPR/Cas9-Mediated Editing of Trinucleotide Repeat Expansion in Myotonic Dystrophy

2017 Molecular Therapy
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CRISPR/Cas9-mediated editing for dominant genetic disorders: efficient excision of trinucleotide repeat expansion in myotonic dystrophy

2017 Human Gene Therapy
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Erratum: Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy (Molecular Therapy (2014) 22 (1614-1624) DOI:10.1038/mt.2014.131)

2015
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"Taming" iPS: Coaxed differentiation of iPS into myogenic precursors using hyperactive transposons expressing the myogenic transcription factor PAX3

2010 Human Gene Therapy
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Refining the tools for gene therapy of hemophilia

2006
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Gene therapy : the next generation - development of improved vector technologies

2005
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Gene delivery to the heart and liver using AAV serotype 9 : implications for cardiovascular disease and hemophilia

2005
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Preclinical and clinical gene therapy for haemophilia

2004 Haemophilia
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Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long-terminal repeats

2003 Haemophilia
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Citations (47)