Innovations and Challenges in Gene and Cell Therapy: From Bench to Bedside

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Background

The field of gene and cell therapy has entered a period of rapid advancement. From CRISPR-Cas9–based genome editing to the clinical application of engineered CAR-T and NK cell therapies, these innovative approaches have demonstrated revolutionary potential in treating cancer, rare genetic disorders, and degenerative diseases. Nevertheless, the field continues to face significant challenges, including dose- and immune-related toxicities, off-target effects, obstacles in large-scale manufacturing, and uncertainties surrounding long-term safety.

This Research Topic aims to capture cutting-edge studies within the domain of gene and cell therapy, focusing particularly on therapeutic innovations and translational hurdles. We especially welcome contributions addressing breakthroughs in delivery systems, immune modulation, and bridging preclinical research with clinical application.

We invite submissions of Original Research articles, Reviews, Mini-reviews, Systematic Reviews, and high-quality Case Reports related to, but not limited to, the following areas:
• Novel gene therapy platforms (viral and non-viral vectors)
• Engineered immune cells (CAR-T, TCR-T, NK-T cells, macrophage-based therapies)
• Synthetic biology and advanced cell-engineering strategies
• Delivery and targeting systems for gene/cell therapies
• Strategies for overcoming host immune responses and resistance mechanisms
• Translational challenges and clinical trial design
• Regulatory considerations, scalability, and manufacturing issues in gene/cell therapy implementation

Through this Research Topic, we aim to foster interdisciplinary dialogue and accelerate the safe, effective, and accessible translation of gene and cell therapies into clinical practice across a broad range of diseases.

Keywords: Gene therapy, Cell therapy, Viral and non-viral vectors, Translational medicine, Immunotherapy

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

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