Gene and Cell Therapy, a Therapeutic Promise for the Future: Successes and Limits

In the last few years, Gene and Cell Therapy research has emerged as one of the most promising technologies for treating several diseases. Gene therapy aims to introduce, edit, modify, or silence specific genes to correct genetic defects using either viral or non-viral approaches. Cell therapy involves the delivery of healthy or engineered cells into the patient’s body with the expectation that the host cells will fuse and repopulate the damaged tissues, improving the clinical phenotype.

Moreover, CAR-T cell therapy offers a promising approach to treat solid tumours and autoimmune diseases, by developing a more targeted and secure system. Considering that recent clinical trials have highlighted limitations and effectiveness of this combined approach, this Research Topic is focused on the progress and future perspectives of applying this technology to the treatment of several genetic disorders including neuromuscular and neurodegenerative diseases, autoimmune disorders, cardiovascular diseases and cancer.

We welcome different article types, such as Original Research, Brief Research Reports, Reviews and Mini Reviews providing new perspectives or novel methods that open further research and discussion to bring significant advances in gene and cell therapy. This Research Topic is breaking new ground in the treatment of a wide range of genetic diseases, and we aim to help change the approach to the treatment of genetic disorders around the world.

We are interested in submissions covering many themes, including but not limited to:

• Clinical and Preclinical studies
• Gene Delivery Optimization, developing more efficient and targeted delivery systems investigating strategies to bypass cellular barriers that prevent efficient gene transfer.
• Novel Gene Editing Technologies, in particular improving the precision of CRISPR-Cas9 and other genome editing tools, reducing off-target effects, and increasing their efficacy in different cell types.
• Enhancement in Cell Therapy Applications, focusing on the development of CAR-based immunotherapies and their possible new applications.
• Investigating the potential use of different types of stem cells (embryonic stem cells, induced pluripotent stem cells, adult stem cells) for tissue regeneration and disease treatment.
• Applications in Specific Genetic diseases to treat monogenic disorders, such as cystic fibrosis, muscular dystrophy, and inherited blood disorders, cancer and neurodegenerative diseases.
• Discussion on Potential Benefits of this approach like personalized medicine and durable effect.
• Ethics and Regulation

Manuscripts describing only bioinformatic analyses of existing databases or bibliographic-based analyses without significant experimental validation will not be accepted to this collection.

Keywords: Stem Cells, Ipscs, CAR-T Cells, Viral Vector, Gene Editing, Non-Viral Approach, CRISP-Cas9, Genetic Disorders, Cancer, 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.