Not finding it? Sign in to also search OpenAlex live.
-
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
204citations -
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
13citations -
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
3citations -
BICC1 Interacts with PKD1 and PKD2 to Drive Cystogenesis in ADPKD2024 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Genetic and Kidney Cyst Diseases Open access
Uyen Tran, Andrew J. Streets, Devon Smith, Annemarie Kirschfink, Lahoucine Izem, Jessie M Hassey, +9 more
3citations -
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
1citations -
Data from: BICC1 interacts with PKD1 and PKD2 to drive cystogenesis in ADPKD2026 DRYAD dataset Biochemistry, Genetics and Molecular Biology Genetic and Kidney Cyst Diseases Open access
Oliver Wessely, Uyen Tran, Andrew J. Streets, Devon Smith, Annemarie Kirschfink, Lahoucine Izem, +8 more
1citations -
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
40citations -
Disease modifying treatment of hereditary transthyretin amyloidosis with polyneuropathy in Germany – expert consensus of the German society of amyloid diseases (DGAK) and the German neurological society (DGN)2026 Neurological Research and Practice article Biochemistry, Genetics and Molecular Biology Amyloidosis: Diagnosis, Treatment, Outcomes Open access
Markus Weiler, Maike Franziska Dohrn, Stefan Gingele, Thomas Skripuletz, Matthias Schilling, Tim Hagenacker, +9 more
2citations -
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
14citations -
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
13citations -
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
1citations -
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 -
Comparative analysis of mouse and human preimplantation development following POU5F1 CRISPR/Cas9 targeting reveals interspecies differences2021 Human Reproduction article Biochemistry, Genetics and Molecular Biology Pluripotent Stem Cells Research Open access
Panagiotis Stamatiadis, Annekatrien Boel, Gwenny Cosemans, Mina Popovic, Bieke Bekaert, Ramesh Reddy Guggilla, +8 more
21citations -
Anti‐C1q autoantibodies may not serve as an adequate biomarker for lung manifestations in systemic sclerosis: a single‐centre, cross‐sectional study2021 British Journal of Dermatology article Medicine Systemic Sclerosis and Related Diseases Open access
Douwe J. Dijkstra, Sophie I E Liem, Nina Marijn van Leeuwen, Cynthia M. Fehres, Jeska Kirsten De Vries-Bouwstra, Leendert Adrianus Trouw
1citations -
P-802 TFAP2C is required for proper segregation of developmental markers regulating trophectoderm commitment during mouse preimplantation development2023 Human Reproduction conference-abstract Biochemistry, Genetics and Molecular Biology Developmental Biology and Gene Regulation Open access
Gwenny Cosemans, Annekatrien Boel, Bieke Bekaert, Dominic Stoop, Susana M. Chuva de Sousa Lopes, Paul J. Coucke, +3 more
0citations -
O-216 CRISPR/Cas9 mediated knock-out (KO) reveals a divergent role for trophectoderm markers GATA2/3 in the mouse and human preimplantation embryo2022 Human Reproduction conference-abstract Biochemistry, Genetics and Molecular Biology Pluripotent Stem Cells Research Open access
Gwenny Cosemans, Annekatrien Boel, Bieke Bekaert, E Pascal, P Stamatiadis, D Stoop, +5 more
1citations