Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders

• Givinostat enhances antisense oligonucleotide efficacy in the mdx52 model of Duchenne muscular dystrophy

  2026 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  0

  citations
• Selector adeno-associated viral vectors facilitate on-target precise genome editing and purge off-target chromosomal insertions

  2026 Trends in biotechnology review Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  0

  citations
• 3D multiscale shape analysis of nuclei and in vivo elastic stress sensors allows force inference

  2025 Biophysical Journal article Biochemistry, Genetics and Molecular Biology Cellular Mechanics and Interactions Open access

  6

  citations
• SUMOylation differentially regulates SMCHD1 complex formation and function in a genomic context-specific manner

  2024 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Genomics and Chromatin Dynamics

  1

  citations
• Selection-free precise gene repair using high-capacity adenovector delivery of advanced prime editing systems rescues dystrophin synthesis in DMD muscle cells

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

  24

  citations
• DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

  2023 RNA Biology article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  2

  citations
• Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

  2023 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent

  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
• Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice

  2022 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  17

  citations
• DMD antisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exon

  2022 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders 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
• In Vitro Delivery of PMOs in Myoblasts by Electroporation

  2022 Methods in molecular biology book-chapter Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  3

  citations
• Broadening the reach and investigating the potential of prime editors through fully viral gene-deleted adenoviral vector delivery

  2021 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access

  44

  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

14 results