Molecular Therapy — Methods & Clinical Development Sign in to save
Aachen papers per year
Papers 17
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Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID2020 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 3 patents
Laura García-Pérez, Marja C.J.A. van Eggermond, Lieke van Roon, Sandra A. Vloemans, Martijn Cordes, Axel Schambach, +16 more
58citations -
Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain2020 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 1 patent
Josse A. Depla, Marina Sogorb-González, Lance Mulder, Vivi Majella Heine, Pavlina S. Konstantinova, Sander J. van Deventer, +4 more
53citations -
Modifying immune responses to adeno-associated virus vectors by capsid engineering2023 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access
Martin Bentler, Romain Hardet, Moritz Ertelt, Daniela Rudolf, Dorota Kaniowska, Andreas Schneider, +8 more
34citations -
Evidence generation and reproducibility in cell and gene therapy research: A call to action2021 Molecular Therapy — Methods & Clinical Development article Medicine Biomedical Ethics and Regulation Open access cited by 1 patent
Mohamed Abou‐El‐Enein, Aris Angelis, Frederick R. Appelbaum, Nancy C. Andrews, Susan Elaine Bates, Arlene S. Bierman, +29 more
25citations -
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector2020 Molecular Therapy — Methods & Clinical Development erratum Medicine Lysosomal Storage Disorders Research Open access cited by 1 patent
Maria Dahl, Emma M. K. Smith, Sarah Warsi, Michael Rothe, Maria J. Ferraz, Johannes M. F. G. Aerts, +7 more
23citations -
AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model2020 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access
Thilo M. Buck, Rogier M. Vos, C. Henrique Alves, Jan Wijnholds
23citations -
Human iPSC-derived preclinical models to identify toxicity of tumor-specific T cells with clinical potential2023 Molecular Therapy — Methods & Clinical Development article Medicine CAR-T cell therapy research Open access
Rosa A. van Amerongen, Laura T. Morton, Umesh Chaudhari, Dennis F.G. Remst, Renate S. Hagedoorn, Cathelijne W. van den Berg, +2 more
13citations -
Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/− retinal organoids2023 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access
Nanda Boon, Xuefei Lu, Charlotte A. Andriessen, M A Orlova, Peter M. J. Quinn, Camiel J. F. Boon, +1 more
11citations -
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease2024 Molecular Therapy — Methods & Clinical Development article Medicine Lysosomal Storage Disorders Research Open access
Jolanda M.P. Liefhebber, Giso Brasser, Elisabeth A. Spronck, Roelof Ottenhoff, Lieke Paerels, Maria J. Ferraz, +6 more
9citations -
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy2024 Molecular Therapy — Methods & Clinical Development article Medicine CAR-T cell therapy research Open access
Isabella Elias Yonezawa Ogusuku, Vera Herbel, Simon Lennartz, Caroline Brandes, Eva M. Argiro, C. Fabian, +12 more
8citations -
AAV6 vectors provide superior gene transfer compared to AAV9 vectors following intramyocardial administration2025 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access
Jianan Wang, Timo Jonker, Aina Cervera-Barea, Zhenyu Dong, Evelien E. Birza, Arnie Boender, +16 more
7citations -
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II2023 Molecular Therapy — Methods & Clinical Development article Medicine Lysosomal Storage Disorders Research Open access
Fabio Catalano, Eva C. Vlaar, Drosos Katsavelis, Zina Dammou, Jeroen C. van den Bosch, Marianne Hoogeveen‐Westerveld, +8 more
6citations -
Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments2025 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access
Basel T. Assaf, Shawn Jay Berens, Brian Lauritzen, Francis Fonyuy Tukov, Cristina Bertinetti‐Lapatki, Manuela Braun, +1 more
3citations -
Ribosome profiling shows variable sensitivity to detect open reading frames for conventional and different types of cryptic T cell antigens2024 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology RNA and protein synthesis mechanisms Open access
Kyra J. Fuchs, Sofia Thomaidou, Arno R. van der Slik, Marian van de Meent, Peter A.C. ’t Hoen, J.H. Frederik Falkenburg, +2 more
1citations
17 results
Most active Aachen authors
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Johannes M. F. G. Aerts 29,390 citations overall2 papers
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Frank J. T. Staal 18,191 citations overall2 papers
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J.H. Frederik Falkenburg 12,934 citations overall2 papers
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Jan Wijnholds 12,369 citations overall2 papers
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Karin Pike‐Overzet 4,627 citations overall2 papers
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Marieke Griffioen 4,156 citations overall2 papers
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Maria J. Ferraz 1,620 citations overall2 papers
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Jacques J. M. van Dongen 55,067 citations overall1 paper
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Peter A.C. ’t Hoen 50,592 citations overall1 paper
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Christine L. Mummery 40,132 citations overall1 paper
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Bart O. Roep 23,802 citations overall1 paper
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Arjan C. Lankester 16,396 citations overall1 paper
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Mirjam van der Burg 14,001 citations overall1 paper
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Camiel J. F. Boon 13,468 citations overall1 paper
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Jaap Jan Zwaginga 12,995 citations overall1 paper