Rare Diseases and Orphan Drugs

It is defined as the study of how the genes and the genome contribute to the different biological processes the research in this field is difficulty studies on a genome wide with the hope of narrowing them down to the list of gene or regions to analyze in detail. The functional genomics is used to the determination of how the individual components of the biological system work can be together to produce the phenotype. these are mainly focus on the expression of gene products in a specific context. A gene therapy is one of the type of medical treatment that involves removing and adding or the changing the person genetic material it is also known as their DNA The gene therapy has the potential of correcting underlying genetic defects and offering the cure rather to managing symptoms.

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