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RNA-based therapies in inherited retinal diseases2022 Therapeutic Advances in Ophthalmology article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 1 patent
Aniz Girach, Isabelle S. Audo, David G. Birch, Rachel M. Huckfeldt, Byron L. Lam, Bart Peter Leroy, +6 more
36citations -
Mechanism of Calcium Permeation in a Glutamate Receptor Ion Channel2023 Journal of Chemical Information and Modeling article Neuroscience Neuroscience and Neuropharmacology Research Open access
Florian Karl Schackert, Johann Biedermann, Saeid Abdolvand, Sonja Minniberger, Chen Song, Andrew J.R. Plested, +2 more
24citations -
A perspective on causality assessment in epigenetic research on neurodegenerative disorders2022 Neural Regeneration Research article Biochemistry, Genetics and Molecular Biology Epigenetics and DNA Methylation Open access
Philippos Koulousakis, Assia Tiane, Niels Hellings, Jos Prickaerts, Daniël van den Hove, Tim Vanmierlo
8citations -
CRISPR-Cas9 induces large structural variants at on-target and off-target sites in vivo that segregate across generations2021 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Ida Höijer, Anastasia Emmanouilidou, Rebecka Östlund, Robin van Schendel, Selma Bozorgpana, Lars Feuk, +3 more
0citations -
High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells2023 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access
Francesca Tasca, Marcella Brescia, Jin Liu, Josephine M. Janssen, Kamel Mamchaoui, Manuel A.F.V. Gonçalves
7citations -
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 -
Production and Storage of High-Capacity Adenoviral Vectors for the Delivery of Advanced CRISPR Systems2026 Methods in molecular biology book-chapter Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access
Xiaoling Wang, Jin Liu, Josephine M. Janssen, Manuel A.F.V. Gonçalves
0citations -
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 -
CRISPR/Cas9-Mediated Introduction of Specific Heterozygous Mutations in Human Induced Pluripotent Stem Cells2021 Methods in molecular biology book-chapter Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access cited by 1 patent
Karina O. Brandão, Catarina Grandela, Loukia Yiangou, Christine L. Mummery, Richard P. Davis
8citations -
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 -
Nanoparticle-mediated mRNA delivery for cancer, autoimmunity, and genetic diseases: a rapid review2026 Frontiers in Drug Delivery article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access
Okechukwu Paul-Chima Ugwu, Fabian Chukwudi Ogenyi, Mariam Basajja, Chinyere Nneoma Ugwu, Mundu M. Mustafa, Michael Ben Okon
5citations