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LEBER CONGENITAL AMAUROSIS DUE TO CEP290 MUTATIONS—SEVERE VISION IMPAIRMENT WITH A HIGH UNMET MEDICAL NEED2021 Retina article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 1 patent
Bart Peter Leroy, David G. Birch, Jacque L. Duncan, Byron L. Lam, Robert K. Koenekoop, Fernanda Belga Ottoni Porto, +2 more
61citations -
EXTH-55. ALL-IN-ONE GENETIC CONSTRUCT TO KNOCKDOWN MIR-21 REDUCES GLIOMA PROLIFERATION IN VITRO2023 Neuro-Oncology article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Lisa Nieland, Anne B. Vrijmoet, Alexandra J E M de Reus, Benjamin Kleinsteiver, Marike L. D. Broekman, Xandra O. Breakefield, +1 more
1citations -
CRISPR/Cas9 Mutagenesis by Translocation of Cas9 Protein Into Plant Cells via the Agrobacterium Type IV Secretion System2020 Frontiers in Genome Editing article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Daan J. Schmitz, Zahir Ali, Chenglong Wang, Fatimah Aljedaani, Paul J. J. Hooykaas, Magdy M. Mahfouz, +1 more
23citations -
Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease2022 Nature Genetics article Biochemistry, Genetics and Molecular Biology Genetic and Kidney Cyst Diseases Open access cited by 1 patent
Yaoxian Xu, Christoph Kuppe, Javier Perales-Patón, Sikander Hayat, Jennifer Kranz, Ali Taleb Abdallah, +33 more
67citations -
Various repair events following CRISPR/Cas9-based mutational correction of an infertility-related mutation in mouse embryos2024 Journal of Assisted Reproduction and Genetics article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Bieke Bekaert, Annekatrien Boel, Andreï Rybouchkin, Gwenny Cosemans, Sofie Declercq, Susana M. Chuva de Sousa Lopes, +5 more
4citations -
PLGA-Nanoparticles for Intracellular Delivery of the CRISPR-Complex to Elevate Fetal Globin Expression in Erythroid Cells2020 Biomaterials article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 1 patent
Luis Javier Cruz, Thamar van Dijk, Olena Vepris, Tracy M.W.Y. Li, Timo Schomann, Fabio Baldazzi, +5 more
49citations -
P-802 Distinct genetic impact of CRISPR/Cas9 gene correction in human embryos compared to induced pluripotent stem cells2022 Human Reproduction article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Bieke Bekaert, Annekatrien Boel, Lisa De Witte, Gwenny Cosemans, Lise Tordeurs, A M De Loore, +6 more
0citations -
Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?2021 Frontiers in Genome Editing article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Stefanie Klaver-Flores, Hidde A. Zittersteijn, Kirsten Canté-Barrett, Arjan C. Lankester, Rob C. Hoeben, Manuel A.F.V. Gonçalves, +2 more
9citations -
Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia2022 Science Translational Medicine article Medicine CAR-T cell therapy research Open access cited by 2 patents
Giorgio Ottaviano, Christos Georgiadis, Soragia Athina Gkazi, Farhatullah Syed, Hong Zhan, Annie Etuk, +52 more
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Divergent Delivery and Expression Kinetics of Lipid and Polymeric Nanoparticles across mRNA Modalities2025 Advanced Science article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access
Irafasha C. Casmil, Josh J. Friesen, Nuthan Vikas Bathula, Anneke Strumpel, Chia-Hao Ho, Ilana Guez, +7 more
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Precision T cell correction platform for inborn errors of immunity2025 Molecular Therapy article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Katariina Mamia, Sólrún Kolbeinsdóttir, Kornel Labun, Zhuokun Li, Anna Zofia Komisarczuk, Salla Keskitalo, +23 more
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High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells2020 Cells article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access
Marcella Brescia, Josephine M. Janssen, Jin Liu, Manuel A.F.V. Gonçalves
30citations -
17th Annual Meeting of the Oligonucleotide Therapeutics Society: A Tribute to Bob Letsinger, Progress in N = 1 Treatments, and Successful First In-Humans CRISPR Trials2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery
Ronald A.M. Buijsen, Aurélie Lacroix, Joseph Ochaba, Daniel O’Reilly, Leonora Abdullahu, Erin M. McConnell
0citations -
Neurodevelopmental Disorder with Dystonia and Chorea Linked to De Novo Variants in the Splicing Regulator SRRM42026 Movement Disorders article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access
Philip Harrer, Volker Kittke, Alice Saparov, Alexej Knaus, Shimriet Zeidler, Rachel Schot, +19 more
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Interferon-driven brain phenotype in a mouse model of RNaseT2 deficient leukoencephalopathy2021 Nature Communications article Immunology and Microbiology interferon and immune responses Open access
Matthias Kettwig, Katharina Ternka, Kristin Wendland, Silvia Zampar, Charlotte Schob, Jonas Franz, +20 more
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The Lrat−/− Rat: CRISPR/Cas9 Construction and Phenotyping of a New Animal Model for Retinitis Pigmentosa2021 International Journal of Molecular Sciences article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access
Céline Koster, Koen van den Hurk, Colby F. Lewallen, Mays Talib, Jacoline B. ten Brink, Camiel J. F. Boon, +1 more
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Mis-localization of endogenous TDP-43 leads to ALS-like early-stage metabolic dysfunction and progressive motor deficits2024 Molecular Neurodegeneration article Medicine Amyotrophic Lateral Sclerosis Research Open access
Yiying Hu, Alexander Hruscha, Chenchen Pan, Martina Schifferer, Michael K. Schmidt, Brigitte Nuscher, +8 more
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Association of bronchial steroid inducible methylation quantitative trait loci with asthma and chronic obstructive pulmonary disease treatment response2022 Clinical and Translational Allergy article Medicine Asthma and respiratory diseases Open access
Elise M. A. Slob, Alen Faiz, Jos van Nijnatten, Susanne J. H. Vijverberg, Cristina Longo, Merve Kutlu, +29 more
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TEAD4 regulates trophectoderm differentiation upstream of CDX2 in a GATA3-independent manner in the human preimplantation embryo2022 Human Reproduction article Biochemistry, Genetics and Molecular Biology Developmental Biology and Gene Regulation Open access
Panagiotis Stamatiadis, Gwenny Cosemans, Annekatrien Boel, Björn Menten, Petra De Sutter, Dominic Stoop, +4 more
37citations