Next-Generation Gene and Cell Therapies: Targeting Diseases at Their Origin

The field of molecular medicine has witnessed rapid progress, especially with the rise of gene and cell therapies that aim to correct diseases at their genetic, cellular, or immunological origins. Unlike conventional therapies that mainly address symptoms, these approaches work by repairing defective genes, modifying immune pathways, or replacing dysfunctional cells. Their application has expanded from rare inherited disorders—such as hemophilia, spinal muscular atrophy, and sickle cell disease—to complex acquired conditions like cancer, autoimmune diseases, and chronic infections.

Breakthroughs in genome-editing tools (e.g., CRISPR-Cas9), delivery systems, and cellular engineering (e.g., CAR-T cells) are driving these advancements. However, challenges such as targeted delivery, long-term efficacy, immunogenicity, and equitable access remain. A deeper understanding of disease pathogenesis at the molecular and cellular level is essential to refine these therapies and ensure safe and effective clinical outcomes.

This Research Topic aims to provide a comprehensive overview of next-generation gene and cell therapies targeting both inherited and acquired disorders. The project seeks to gather and present the latest research that addresses key mechanisms underlying genetic mutations, immune system dysfunction, and cellular abnormalities. By highlighting innovative therapeutic technologies and clinical approaches, the goal is to advance scientific knowledge and clinical translation in the field.

Furthermore, this initiative encourages cross-disciplinary collaboration among experts in molecular biology, immunology, regenerative medicine, and bioengineering. Ultimately, the goal is to accelerate the development of precise, safe, and durable therapies that can transform patient care and potentially offer curative solutions.

The Research Topic will cover a wide range of gene and cell therapy approaches aimed at treating both rare and common disorders. Submissions may explore advanced gene-editing techniques, novel vector platforms, and cellular therapies such as CAR-T- and stem cell-derived interventions. Studies addressing inherited diseases like thalassemia and muscular dystrophies, as well as acquired conditions such as cancer, autoimmune syndromes, and viral infections, are welcome.

Special emphasis will be placed on translational research, safety and efficacy data, immune response modulation, delivery system optimization, and ethical and regulatory considerations. Reviews, original research, and perspectives that discuss manufacturing challenges, long-term monitoring, and equitable access will also be prioritized. This Research Topic aims to foster innovation and collaboration across disciplines to enhance the therapeutic potential and global impact of gene and cell therapies.

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Review
• Study Protocol
• Systematic Review
• Technology and Code

Keywords: Gene Therapy, Cell Therapy, Inherited Disorders, CRISPR, CAR-T Cells, Immunotherapy, Regenerative Medicine, Rare Diseases, Translational Research, Genomic Medicine

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.