Now a days it is being focused on the functionality and the speciality of genes to use them as precursor of antipeptides, so in case of any disorder no need to give antipeptides externally. Gene therapy is an emerging technique in which genes are preferred to treat or prevent the disorders inspite of using drugs and surgery’s. Researchers are testing possible approaches to gene therapy including,Replacing a mutated or defective gene that causes disease with a healthy copy of the gene, Inactivating, or “knocking out,” a mutated gene that is functioning improperly,Introducing a new gene which produces the anti effectives into the body to help fight a disease. Although gene therapy is a promising treatment option for a number of diseases like inherited disorders, some types of cancer, and on certain viral infections, But this technique remains risky and is still under study to make sure that it will be safer and effective. Gene therapy is currently being tested only for diseases that have no other cures.
INTRODUCTION
Genes are the basic inheritance and regulatory molecules which commands the cell via expressing themselves in terms of proteins. Therapy with using genes is a novel treatment method in which genes or short oligonucleotide sequences used as therapeutic molecules, instead of using conventional drug compounds and surgery’s . This technique is widely used to treat those defective genes which are responsible in the development of diseases [1,2]. The treatment is better and preferred over conventional drugs methods [2]. Our genetic program is made up of thousand of genes, stretches of DNA, that generally code for different proteins that do particular jobs in the cells in our body. The idea of using drugs and antipeptides is replaced by gene therapy, as the genes express them selves in terms of peptides [3].
HISTORICAL ASPECTS
The first idea of gene therapy was introduced by Theodore Friedmann and Richard Roblin during 1960’s. They are the first who gave the concept of treatment via introducing a particular gene which lasts to the next generations [4]. Now we have several gene editing and inserting tools e.g: CRISPR-CAS9, which may be useful in cancer and viral treatments.
WHY GENE THERAPY!
The research and the exploration of inherent molecules such as RNA and DNA gave rise to the concept of treating disorders and syndromes directly by an organism itself for life time. As the inherent molecules have the capability to transfer generation over generation so transferring the gene of interest is permanent cure for the syndromes and disorders.
- The genes have the ability to inherit in next generation thus leading to permanent cure for the disorders and syndromes.
- Gene are the regulatory molecules present in cell so the defective ones can be removed which results in the permanent cure with no further medication.
REFFERNCES
- Bainbridge, J. W. B.; Smith, A. J.; Barker, S. S.; Robbie, S.; Henderson, R.; Balaggan, K.; Viswanathan, A.; Holder, G. E. et al. (2008). "Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis". New England Journal of Medicine 358 (21): 2231–2239.
- Genetic Science Learning Center. (2012, December 1) What is Gene Therapy?. Retrieved January 30, 2018, from http://learn.genetics.utah.edu/content/genetherapy/intro/
- Shalini Jaiswal*, 2Gautam Singh, Jawwad Husain2 1Assistant Professor, Chemistry Department, AMITY University, Noida Campus, India. 2Biotechnology Department, AMITY University, Noida Campus, India. *Corresponding author’s E-mail: shaliniajaiswal@gmail.com “Mechanism and Antimicrobial Application of Histatin 5, Defensin and Cathelicidin Peptides Derivatives”
- Theodore Friedmann and Richard Roblin "Gene therapy for human genetic disease?" 1972 Mar 3;175(4025):949-55
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