Biological Research and the Drug Development of Rare Genetic Disorders

Abstract

There are many cases of rare genetic disorders in the United States.  Millions of people in the United States are affected by approximately 7,000 rare diseases. Are researchers and their studies doing well on the knowledge of this? There actually is a lot of research on how the genetic disorders occur and how and what is being done to not exactly cure the disease, but to stabilize the rare occurrence. As mentioned, a lot of research has been done and is still going on. With that being said, drug discovery has grown rapidly. Examples of drug repurposing and some explanations on how it is done, is given. As well as the pros and the cons of the target-based screens are discussed. Reflection about biologics will be given, such that about recombinant and gene therapy, along with autologous transplant. The type of disease models used and the role biomarkers have in drug discovery will be taken in consideration.