gene editing

A-to-I edited miRNA-99a-5p in bone marrow mesenchymal stem cell-derived exosomes promotes fracture healing by regulating GDF11

Advances in Exosome Engineering for Cardiovascular Applications

Exosomes have emerged as favorable natural carriers of theragnostic molecules. Once considered the “disposal bags of the cells” for cellular metabolic waste, they have emerged as central players in intercellular communication. They carry bioactive-molecule cargo from donor cells to recipient cells, which respond to the...

Machine learning-driven DNA mutation detection and CRISPR gene editing visualization techniques

THE EL-RAKHAWI PRE-CLINICAL PHOTONIC-BIO VERIFICATION SYSTEM FOR CRISPR GENE EDITING

The El-Rakhawi Pre-Clinical Photonic-Bio Verification System for CRISPR (DOI: 10.5281/zenodo.23507507) addresses the critical safety bottleneck in CRISPR gene editing: off-target effects that can cause oncogenic mutations. This PCT-ready patent specification discloses a revolutionary lab-on-a-chip architecture replacin...

PMCID: PMC13557426; Supplemental Data (Post-euth. Data)

Supplemental data for the manuscript, Toward Next-Gen Cell Therapy for Pediatric Patients: Neonatal Hepatocytes Tolerate Electroporation-Mediated Gene Editing and Engraft in the Liver, 2026.

Genetic Engineering for beta-Thalassemia

Genetic Engineering for Beta-Thalassemia Description Genetic Engineering for Beta-Thalassemia: Molecular Mechanisms, Gene Therapy, Genome Editing, and Future Therapeutic Strategies explores the scientific foundations and emerging therapeutic applications of genetic engineering for beta-thalassemia, an inherited blood d...

PMCID: PMC13557426; Supplemental Data (Post Euth. Data_3)

Supplemental data for the manuscript, Toward Next-Gen Cell Therapy for Pediatric Patients: Neonatal Hepatocytes Tolerate Electroporation-Mediated Gene Editing and Engraft in the Liver, 2026.

Precision Cell Fate Engineering with CRISPR Epigenetic Editing for Regeneration and Off-the-Shelf Immunotherapy

Induced pluripotent stem cell (iPSC)-based regenerative medicine and cellular immunotherapy are severely hindered by heterogeneous cell phenotypes, unstable cellular functions and inconsistent large-scale manufacturing. Different from traditional CRISPR/Cas9 genome editing that induces DNA double-strand breaks and geno...

PMCID: PMC13557426; Supplemental Data (Ploidy & Transplant Day)

Supplemental data for the manuscript, Toward Next-Gen Cell Therapy for Pediatric Patients: Neonatal Hepatocytes Tolerate Electroporation-Mediated Gene Editing and Engraft in the Liver, 2026.

PMCID: PMC13557426; Supplemental Data (Post Euth. Data_2)

Supplemental data for the manuscript, Toward Next-Gen Cell Therapy for Pediatric Patients: Neonatal Hepatocytes Tolerate Electroporation-Mediated Gene Editing and Engraft in the Liver, 2026.

Genetic Engineering for Diabetes: From Recombinant Insulin to Genome-Edited Beta-Cell Replacement

Genetic Engineering for Diabetes explores the scientific principles, emerging technologies, and potential applications of genetic engineering in the prevention, understanding, and treatment of diabetes mellitus. The work introduces the genetic and molecular mechanisms associated with diabetes, including the roles of in...

Ferro-aging and ex vivo venetoclax resistance in AML: limited incremental information beyond lineage and BCL2-family programs

Reproducibility materials accompanying the revised manuscript "Ferro-aging tracks ex vivo venetoclax resistance and BCL2-family transcriptional state in AML." The archive contains nine analysis components, complete S1–S14 supplementary workbooks, a cross-wave incremental-evaluation workbook, all 26 CSV detail files, fi...