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

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 edit, introduce, modify, or silence specific genes in order 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 to restore, replace or regenerate damaged tissues, thereby improving clinical outcomes. Recently, new approaches in cell and gene therapy have shown the potential of non-viral delivery systems like lipid nanoparticles encapsulating mRNAs. These innovations aim to make treatments safer and more available for many diseases. Different nano-carriers encapsulate and transport genetic cargo into specific human cells without relying on traditional viral vectors. At the same time, new automated platforms standardize cell production in closed systems to minimize the contamination risks and the costs of preparing cell products.These innovations aim to make treatments safer and available for many diseases. Different nano-carriers encapsulate and transport genetic cargo into specific human cells without relying on traditional viral vectors. At the same time, new automated platforms standardize cell production in closed loops to minimize the contamination risks and the costs of preparing cell products.Considering that recent clinical trials have highlighted the 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, cancer and diabetes.We welcome Original Research, Brief Research Reports, Reviews and Mini Reviews that advance the field of gene and cell therapy through innovative discoveries, novel methodologies and thought-provoking perspectives. Research in gene and cell therapy is transforming the treatment landscape for a broad range of genetic diseases. Our aim is to show the development and clinical translation of innovative therapeutic strategies that improve outcomes for patients worldwide.We welcome submissions on a broad range of topics, 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 - Nano-carrier Technology and Applications, with a focus on efficient and safe delivery; including the protection for fragile genetic material, lowering immune reactions, and allowing precise target delivery- Advances in Cell Therapy Applications, including the development of CAR-T cell therapy and its emerging clinical applications, as well as the potential use of different types of stem cells (embryonic stem cells, induced pluripotent stem cells, adult stem cells) for regenerative medicine and disease treatment.- Applications in Specific Genetic diseases to treat monogenic disorders, such as cystic fibrosis, haemophilia, muscular dystrophy, and inherited blood disorders, cancer and neurodegenerative diseases.- Discussion on Potential Benefits of these approaches for personalized and precision medicine.- Regulatory and Ethical Considerations for emerging gene and cell therapies.Manuscripts describing only bioinformatic or bibliographic analyses of existing databases without significant experimental validation will not be accepted to this collection. Topic editors: Mirella Meregalli, Marzia Laura Chiara Belicchi, Silvia Erratico Source: https://www.frontiersin.org/research-topics/84698/gene-and-cell-therapy-a-therapeutic-promise-for-the-future-successes-and-limits-volume-ii