ABSTRACT
Genetic disease is characterized as an ailment due to a defect in an individual’s genome, chromosomal abnormalities, and gene mutations. A successful therapeutic and diagnostic intervention on genetic diseases has been unrevealed predominately, and it demands technological development. Nanomedicine is a boon in various fields of medical applications from the development of diagnostic devices, contrast agents, in vivo imaging, analytical tools to drug delivery vehicles. Versatilities of nanomedicines owing to the dimension, biocompatibility, and manipulating capability to achieve successful delivery at the targeted site of action and their lower toxicity have made this field a technological revolution to combat different unmet needs in health. This is a possible alternative for combating chronic genetic disorders by targeting a particular gene(s) or tissue or cell, which creates a pathological state. Thus, nanomedicine could be a potential therapeutic tool to treat different genetic pathological conditions, such as diabetes, heart diseases, lysosomal storage disorders, Alzheimer’s disease, cystic fibrosis, etc. In this chapter, we will discuss several strategies to manipulate or knock down the expression of the lethal gene(s) specifically responsible for some of such diseases. Here we mainly focus on diseases 104such as cancer and its fate while manipulating genetic alteration by the nanomedicinal strike.
