RNA-programmed genome editing in human cells

• Retinal Ciliopathies and Potential Gene Therapies: A Focus on Human iPSC-Derived Organoid Models

  2024 International Journal of Molecular Sciences article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

  12

  citations
• Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants

  2023 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  19

  citations
• Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells

  2022 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  21

  citations
• Human Induced Pluripotent Stem Cell-Based Modelling of Spinocerebellar Ataxias

  2021 Stem Cell Reviews and Reports book-chapter Biochemistry, Genetics and Molecular Biology Pluripotent Stem Cells Research Open access

  17

  citations
• A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

  2021 Frontiers in Genome Editing article Medicine Hemoglobinopathies and Related Disorders Open access cited by 4 patents

  19

  citations
• 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

  9

  citations
• Novel Therapeutic Approaches for the Treatment of Retinal Degenerative Diseases: Focus on CRISPR/Cas-Based Gene Editing

  2020 Frontiers in Neuroscience article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 3 patents

  23

  citations
• A genome-wide CRISPR/Cas phenotypic screen for modulators of DUX4 cytotoxicity reveals screen complications

  2020 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  2

  citations
• Genetic Screening for Novel Regulators of Immune Checkpoint Molecules

  2020 Trends in Immunology review Medicine Cancer Immunotherapy and Biomarkers

  5

  citations
• A primer to gene therapy: Progress, prospects, and problems

  2020 Journal of Inherited Metabolic Disease article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 4 patents

  27

  citations
• Adenoviral Vectors Meet Gene Editing: A Rising Partnership for the Genomic Engineering of Human Stem Cells and Their Progeny

  2020 Cells article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  34

  citations
• Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components

  2020 Gene Therapy article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access cited by 1 patent

  66

  citations

12 results