Viral vector platforms within the gene therapy landscape

• AAV-mediated gene replacement therapy for LRAT-associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model

  2026 Gene Therapy article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders

  0

  citations
• Defining core competencies for advanced therapy medicinal products translation

  2026 Frontiers in Digital Health article Medicine Biomedical Ethics and Regulation Open access

  2

  citations
• Current capacity and gaps in viral vector manufacturing: a survey of centers associated with CTIWP of EBMT, NXTGEN-hightech, DARE-NL, T2EVOLVE, and GoCART coalition

  2025 Bone Marrow Transplantation article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  0

  citations
• AAV6 vectors provide superior gene transfer compared to AAV9 vectors following intramyocardial administration

  2025 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  7

  citations
• RNA Interference Targeting Small Heat Shock Protein B8 Failed to Improve Distal Hereditary Motor Neuropathy in the Mouse Model

  2025 The Journal of Gene Medicine article Neuroscience Hereditary Neurological Disorders

  2

  citations
• Monitoring and modulating cardiac bioelectricity: from Einthoven to end-user

  2024 EP Europace article Medicine Cardiac electrophysiology and arrhythmias Open access

  7

  citations
• CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice

  2024 Molecular Therapy article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  9

  citations
• The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

  2024 Nature Reviews Drug Discovery review Medicine Biomedical Ethics and Regulation

  41

  citations
• STRAIGHT-IN: a platform for rapidly generating panels of genetically modified human pluripotent stem cell lines

  2024 Nature Protocols article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering

  12

  citations
• Non‐coding RNAs to treat vascular smooth muscle cell dysfunction

  2024 British Journal of Pharmacology article Biochemistry, Genetics and Molecular Biology MicroRNA in disease regulation Open access

  15

  citations
• Sonogenetics for Monitoring and Modulating Biomolecular Function by Ultrasound

  2024 Angewandte Chemie International Edition article Engineering Ultrasound and Hyperthermia Applications Open access

  48

  citations
• Sonogenetics for Monitoring and Modulating Biomolecular Function by Ultrasound

  2024 Angewandte Chemie article Engineering Ultrasound and Hyperthermia Applications Open access

  1

  citations
• Advanced Therapy Medicinal Products: Clinical, Non-clinical, and Quality Considerations

  2024 Springer eBooks book-chapter Medicine Biomedical Ethics and Regulation

  0

  citations
• Impact of the Membrane Structure of the Stationary Phase on Steric Exclusion Chromatography (SXC) of Lentiviral Vectors

  2023 Membranes article Biochemistry, Genetics and Molecular Biology Protein purification and stability Open access

  2

  citations
• Reversed Phase-Liquid Chromatography for Recombinant AAV Genome Integrity Assessment

  2023 Analytical Chemistry article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 1 patent

  1

  citations
• Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies

  2023 International Journal of Molecular Sciences article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

  123

  citations
• Scaling Up of Steric Exclusion Membrane Chromatography for Lentiviral Vector Purification

  2023 Membranes article Biochemistry, Genetics and Molecular Biology Protein purification and stability Open access cited by 2 patents

  9

  citations
• Adenoviral vectors for cardiovascular gene therapy applications: a clinical and industry perspective

  2022 Journal of Molecular Medicine article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  39

  citations
• Gene therapy: Comprehensive overview and therapeutic applications

  2022 Life Sciences article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research cited by 1 patent

  339

  citations
• Particles in Biopharmaceutical Formulations, Part 2: An Update on Analytical Techniques and Applications for Therapeutic Proteins, Viruses, Vaccines and Cells

  2021 Journal of Pharmaceutical Sciences article Biochemistry, Genetics and Molecular Biology Protein purification and stability Open access

  43

  citations

20 results

• Non-Human Primate-Derived Adenoviruses for Future Use as Oncolytic Agents?

  2020 International Journal of Molecular Sciences article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 1 patent

  25

  citations

1 result