Recent Advances in Gene Therapy

Veena Priyadarshini^*
*Correspondence: Veena Priyadarshini, Department of Life Sciences, B.S. Abdur Rahaman Crescent Institute of Science and Technology, Chennai, Tamil Nadu, India, Email:

Commentary

Gene delivery systems are vital for treating human genetic illnesses with gene therapy. Gene therapy is a one-of-a-kind method of curing any ailment by using an adaptable gene. Gene therapy is a promising treatment option for a variety of ailments, including hereditary disorders, viral infections, and cancer. The effective effects of gene delivery systems are dependent on the ability to target gene delivery systems in a variety of ways. For the practical use of gene therapy, certain viable gene delivery techniques have recently been reported. Although gene therapy is a promising treatment option for a variety of diseases, including cancer, hereditary abnormalities, and certain viral infections, it is still a dangerous procedure that is being studied to ensure its safety until it is effective. Gene therapy is currently being researched only for disorders that have no other treatment options. To activate gene expression, genetic molecules reach the nuclei of host cells. Gene therapy was developed to send genetic information to a patient's somatic cells to produce specific therapeutic proteins to control genetic disorders. To build an effective gene delivery system, a thorough understanding of the interaction between the targeting cell and the gene delivery system is essential [1].

A plasmid-based gene expression system that controls the function of a gene within the targeting cell, a gene that encodes a specific therapeutic protein, and a gene delivery system that controls the delivery of the gene expression plasmid to a specific location within the body are the three components of gene delivery systems. The foreign genetic material must remain stable within the host cells for the gene delivery method to work. Virus-based vectors first appeared in the 1980s as a technique for transgene expression. The vaccinia virus was first employed as a vaccine vector in 1984 to protect chimpanzees against Hepatitis B. The non-viral gene delivery technology was the first to demonstrate cellular phenotypic change in the body via DNA exposure. Since the discovery of DNA as the basic unit of heredity, the ability to make local alterations in the human genome has been a goal of medicine. Gene therapy is defined as the ability to improve genes by correcting altered (mutated) genes or site-specific alterations with therapeutic treatment as the goal. Following that, various tactics that are commonly employed for this aim are outlined. Currently, gene therapy is primarily practiced in research facilities, and its applicability is still in its early stages. [2,3].

Recombinant DNA technology is a process in which a gene of interest or a healthy gene is put into a vector, which can be plasmidial, nanostructured, or viral; the latter is the most commonly utilised due to its efficiency in infiltrating cells and introducing genetic material. Despite the success of some protocols, the gene therapy process remains difficult, and many approaches require further research. It is necessary to identify and reach the precise bodily cells that require treatment. A method for efficiently distributing gene copies to cells must be developed, and diseases and their rigid genetic ties must be fully comprehended. There's also the question of gene therapy's target cell type, which is currently split into two categories: germline gene therapy and somatic cell gene therapy. Germline gene therapy involves introducing functional genes into the genome of stem cells, such as sperm and eggs. The changes are passed on through the generations. Gene therapy involves the insertion of a normal gene into the genome to replace an aberrant gene that causes a disease. The difficulty in releasing the gene into the stem cell is one of the most critical hurdles in the process. Thus, a molecular carrier known as a "vector" is used to release the gene, which must be very specific, efficient in the release of one or more genes of the sizes required for clinical applications, immune system-unfriendly, and purified in large quantities and high concentrations in order to be produced and made available on a large scale.

Once put into the patient, the vector should not cause allergic reactions or inflammation; instead, it should improve normal functioning, rectify deficits, or prevent harmful activities. Because of their remarkable potential for longevity and self-renewal, hematopoietic stem cells have become ideal targets for gene transfer. The manufacture of gene transfer vectors for the generation of induced Pluripotent Stem cells (iPS) in order to promote differentiation of the iPS and afford an extra phenotype from this differentiated derived cell is one example of this combination of gene therapy and stem cells. Patients who require a liver transplant due to chronic liver disease and hepatitis virus infection (e.g., hepatitis B virus and hepatitis C virus) are likely to get mature hepatocytes or those produced from iPS cells.

RNA-dependent polymerase complexes and negative-strand RNA templates are provided by the sophisticated system. In patients undergoing transplantation for AIDS-related lymphoma, RNA-based gene delivery systems were used to transfer HIV using lenti-viral vectors-modified CD 34(+) cells. Oncolytic viruses (OVs) are a new type of treatment for cancer-related disorders. They've now shifted their focus to cancer research, with the goal of improving their therapeutic treatment potential. For the interaction of OVs and tumour cells, the positive and negative impacts of the plethora for their alteration have been discussed to boost their infectivity, anti-tumor immunity, and therapeutic safety. Through differentiation of T cells expressing IL-12R2 or IL-18R, an oncolytic adenovirus co-expressing IL-12 and IL-18 increases tumor-specific immunity [4,5].

References

1. Gonçalves, Giulliana Augusta Rangel, and Raquel de Melo Alves Paiva. "Gene therapy: advances, challenges and perspectives." Einstein (Sao Paulo) 15 (2017): 369-375.

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3. Vaughan, Hannah J., and Jordan J. Green. "Recent advances in gene therapy for cancer theranostics." Curr Opin Biomed Eng 20 (2021): 100300.

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4. Goswami, Reena, Gayatri Subramanian, Liliya Silayeva, and Isabelle Newkirk et.al "Gene therapy leaves a vicious cycle." Front Oncol(2019): 297.

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5. Bulaklak, Karen, and Charles A. Gersbach. "The once and future gene therapy." Nat Comm 11(2020): 1-4.

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