Published Paper Details:

Genetic diseases present a major threat to human health and have long been difficult to tackle. Fortunately, there are promising gene therapy strategies, such as siRNA, shRNA, antisense oligonucleotides, CRISPR/Cas9, plasmid DNA, and miRNA, which hold significant potential in biomedical applications. However, to maximize the effectiveness of these therapies, it is essential to create advanced drug delivery systems that can protect these therapeutic agents from degradation in the body and accurately target specific tissues, cells, and even organelles. In conclusion, developing efficient drug delivery vehicles is vital for progressing gene therapy and addressing genetic disorders. As one of the most significant topics of the 21st century, gene therapy offers the promise of cures for many diseases, raises ethical debates about altering human traits, and introduces a form of medical treatment that many once deemed unimaginable. However, conducting human gene therapy comes with various concerns. This review article explores the background of gene therapy, including its introduction, the nature of genetic diseases, gene function, germline gene therapy, the challenges faced, and the various approaches employed. It covers ex vivo, in vitro, and in vivo gene therapies, along with the associated risks. The review addresses a wide range of topics, from the historical context and societal relevance of gene therapy to the latest advancements in the field, including research and developments in India.