Gene therapy: A progress in childhood disease

Dewase, Dhaval Shriramji and Narwade, Anuradha R (2025) Gene therapy: A progress in childhood disease. GSC Biological and Pharmaceutical Sciences, 31 (1). pp. 319-337. ISSN 2581-3250

As gene therapy is one of the hottest topics of the new century, it carries the excitement of a cure to most of dis controversy surrounding the altering of human imperfection, and the promise of a type of medical treatment most of cause would never imagine possible The recent sequence of the human genome combined with the development of massively high throughput genetic analysis technologies is driving unprecedented growth in knowledge of the molecular basis of disease. While this has already had a major function in our diagnostic power, the therapeutic benefits remain largely unrealized. This review examines progress in the exciting and challenging form of gene therapy, in particular we focus on the treatment of genetic disease in infants and children where the most significant successes have been observed to date, despite the majority of total participants coming from adults. Notably, gene transfer to the haematopoietic compartment has provided the clearest examples of therapeutic benefit, particularly in the context of primary immunodeficiencies. (1) Material and methods: A thorough search of literature carried out through the National library of medicine (PubMed), SCOPUS, EMBASE databases using different Keywords. All the relevant articles were analyzed according to their importance And reviewed to evaluate the past present and future perspective of gene therapy. Resalty: With a possibility to eliminate and present AIDS, malignancies, hereditary disorders and it's conceivable cure for cardiac disorders, gene therapy is nothing short of a medical phenomenon. (1)