Gene Therapy Technology: Advances, Challenges and Perspectives
A special issue of Applied Sciences (ISSN 2076-3417). This special issue belongs to the section "Applied Biosciences and Bioengineering".
Deadline for manuscript submissions: closed (15 April 2022) | Viewed by 500
Special Issue Editors
Interests: missense/nonsense mutations; ribosome readthrough; rare diseases; albumin fusion proteins
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Mutations affecting coding and non-coding gene regions result in the alteration of the normal flow of genetic information at different levels. The knowledge of disease mechanisms paved the way for the development of tailored correction approaches. Among these, the ability to repair or to add a functional copy of the defective gene has been an objective in medicine since the recognition of the gene as the basic unit of heredity. Thus, gene therapy has been considered the holy grail for the treatment of inherited diseases. This approach became possible through the advances of genetics and bio-engineering that enabled the manipulation of viral vectors for the delivery of extra-chromosomal material to target cells. The viruses are more often investigated due to their efficiency of invading cells and inserting their genetic material. However, there is great concern regarding exacerbated immune responses toward the injected vectors and genome manipulation, especially in germ line cells.Moreover, some limitations (i.e neutralizing antibody, tissue tropism, loading capacity) of viruses boosted the research in the development of alternative non-viral delivery strategies that are currently under investigation, but far for being optimized.
Overall, despite in vivo studies showed satisfactory results with approved protocols in clinical trials for various inherited diseases, gene therapy technology still needs supporting experimental evidences, with lights and shadows that need further investigation/optimization.
Dr. Alessio Branchini
Dr. Dario Balestra
Guest Editors
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Keywords
- Gene therapy
- viral vectors
- gene delivery
- genome/base editing
- cell therapy
