In vivo genome editing using Staphylococcus aureus Cas9

• Rewriting the script: gene therapy and genome editing for von Willebrand Disease

  2025 Frontiers in Genome Editing article Medicine Hemophilia Treatment and Research Open access

  3

  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
• Gene Editing for the Treatment of Hypercholesterolemia

  2024 Current Atherosclerosis Reports article Medicine Lipoproteins and Cardiovascular Health Open access cited by 1 patent

  25

  citations
• One-step generation of tumor models by base editor multiplexing in adult stem cell-derived organoids

  2023 Nature Communications article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access cited by 1 patent

  65

  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
• Additional file 2 of A new compact adenine base editor generated through deletion of HNH and REC2 domain of SpCas9

  2023 Figshare dataset Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  0

  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
• 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

10 results