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Gene therapy in paediatrics – A review

Publisher : International Journal of Pharmaceutical Sciences Review and Research

Campus : Kochi

School : School of Pharmacy

Department : Pharmaceutics

Year : 2015

Abstract : Genes are the basic physical and functional units of hereditary and are the specific sequences of bases that are carried on chromosomes which encode instruction, ensure the formation of proteins. Deficiency or disorders of genes in cells leads to the genetic disorders resolved with the respected treatment of gene therapies. The different types of viruses used as gene therapy are retroviruses, adenoviruses, adeno-associated viruses, herpes simplex viruses. Gene therapy is a type delivery where therapy carries the promise of cures for many diseases and for types of medical treatment most of us would not have thought possible. With gene therapy, the treatment or elimination of inherited diseases or physical conditions due to these mutations could become a reality. Gene therapy methods have continued to develop rapidly, and many initial limitations that hampered clinical application have been overcome. Thus serious consideration of clinical application of gene therapy is warranted for selected disorders in which the pathogenesis is well defined. Gene therapy is used to pediatrics for treating leukaemia, diabetics, arthritis, cystic fibrosis etc.

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