Ermira Samara-Kuko

Vrije Universiteit Brussel — Belgium

Biochemistry, Genetics and Molecular Biology · Life Sciences

11h-index1.5kcitations23works2.02yr avg
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Research Topics

CRISPR and Genetic Engineering(16), Virus-based gene therapy research(13), RNA Interference and Gene Delivery(8), Muscle Physiology and Disorders(3), Neurogenetic and Muscular Disorders Research(3)

Publications23 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
Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy
Molecular Therapy·2022· 70 citedOpen Access
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk
UNC Libraries·2020Open 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
Next-generation muscle-directed gene therapy by in silico vector design
Nature Communications·2019· 56 citedOpen Access
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9
Molecular Therapy·2018· 63 citedOpen Access
120. Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9
Molecular Therapy·2016Open Access
628. Transposons Expressing Full-Length Human Dystrophin Enable Genetic Correction of Dystrophic Mesoangioblasts and iPS-Derived Mesoangioblast-Like Cells
Molecular Therapy·2016Open Access
<i>piggyBac</i>transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts
Nucleic Acids Research·2015· 26 citedOpen Access
Next-generation muscle-directed gene therapy using skeletal-muscle specific transcriptional modules identified by genome-wide computational analysis
VUBIR (Vrije Universiteit Brussel)·2015
Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy
Molecular Therapy·2014· 44 citedOpen Access
Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates
Molecular Therapy·2014· 77 citedOpen Access
Overcoming the challenges in gene therapy for haemophilia
UCL Discovery (University College London)·2013
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk
Hepatology·2011· 136 citedOpen Access
"Taming" iPS: Coaxed differentiation of iPS into myogenic precursors using hyperactive transposons expressing the myogenic transcription factor PAX3.
VUBIR (Vrije Universiteit Brussel)·2010
Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation  
Stem Cells·2010· 44 citedOpen Access
Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Non-Viral Paradigm for Coaxed Differentiation
VUBIR (Vrije Universiteit Brussel)·2010· 19 cited
Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates
Nature Genetics·2009· 975 cited
Sustained therapeutic factor IX expression in liver using integration-deficient lentiviral vectors.
VUBIR (Vrije Universiteit Brussel)·2009· 1 cited
Novel enhanced transposases result in robust stable gene transfer into hematopoietic, mesenchymal and muscle progenitor/stem cells
VUBIR (Vrije Universiteit Brussel)·2008
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Frequent Co-authors

Thierry VandenDriessche(23), Marinee Chuah(22), Janka Mátrai(14), Abel Acosta‐Sanchez(10), Melvin Y. Rincón(9), Ling Ma(7), Lajos Mátés(6), Zoltán Ivics(6), Pieter De Bleser(6), Shilpita Sarcar(6), Maurilio Sampaolesi(6), Eyayu Belay(5), Zsuzsanna Izsvák(5), Nisha Nair(5), Mario Di Matteo(5), George Dickson(5), Alessio Cantore(4), Liesbeth De Waele(4), Luigi Naldini(4), Hanneke Evens(4)