Rationally engineered Cas9 nucleases with improved specificity

• Precision genome editing using combinatorial viral vector delivery of CRISPR-Cas9 nucleases and donor DNA constructs

  2024 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access cited by 1 patent

  15

  citations
• Generating high-fidelity cochlear organoids from human pluripotent stem cells

  2023 Cell stem cell article Neuroscience Hearing, Cochlea, Tinnitus, Genetics Open access cited by 3 patents

  73

  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
• High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells

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

  7

  citations
• CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies

  2022 WIREs Mechanisms of Disease article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent

  23

  citations
• Modulating CRISPR-Cas Genome Editing Using Guide-Complementary DNA Oligonucleotides

  2022 The CRISPR Journal article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  0

  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
• HideRNAs protect against CRISPR-Cas9 re-cutting after successful single base-pair gene editing

  2022 Scientific Reports article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  4

  citations
• SDR enzymes oxidize specific lipidic alkynylcarbinols into cytotoxic protein-reactive species

  2022 eLife article Pharmacology, Toxicology and Pharmaceutics Bioactive Compounds and Antitumor Agents Open access

  10

  citations
• Modulating CRISPR-Cas genome editing using guide-complementary DNA oligonucleotides

  2022 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  0

  citations
• Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy

  2021 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access cited by 2 patents

  23

  citations
• Precise and broad scope genome editing based on high-specificity Cas9 nickases

  2020 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  36

  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 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
• High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells

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

  30

  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

16 results