CNS specific antisense oligonucleotide plays an important role in modern healthcare, as it acts on the messenger RNA (mRNA) level before the protein is made to inhibit expression of certain diseases-causing genes. Antisense oligonucleotides are single-stranded oligodeoxynucleotides that can mutate the RNA and modify protein expression. They have the potential to target any gene product of interest. It is a single-stranded sequence complementary to the sequence of the target gene's transcribed messenger RNA (mRNA) within a cell.
CNS specific antisense oligonucleotide is used as a therapeutic agent to treat neurodegenerative disorders. It helps in preventing the development of disease by blocking the metabolic pathway of a particular protein. Moreover, antisense oligonucleotides mediated therapies or treatments target the source of pathogenesis and have a higher chance of success than treatments targeting downstream pathways. These drugs are used to treat central nervous system disorders, as they have specific molecular targeting and extended pharmacological properties.
CNS specific antisense oligonucleotide is often used to treat neurodegenerative disorders and associated rare diseases. Thus, with the increasing prevalence of neurodegenerative disorders or central nervous system (CNS) disorders around the world, the use of or demand for CNS specific antisense oligonucleotide is also increasing with a rapid pace. Neurological disorders are diseases of the central/peripheral nervous system. CNS diseases are a group of neurological disorders that affect the structure or function of the brain or spinal cord.
Alzheimer's is the sixth-leading cause of death in the United States. According to the Centers for Disease Control and Prevention (CDC), in 2020, around 5.8 million people in the U.S. were living with Alzheimer's disease, and the number is expected to nearly triple to 14 million people by 2060. The number of people living with Alzheimer's disease doubles every five years. CNS specific antisense oligonucleotide paved the way toward translating their use into therapies for various genetic and neurological conditions.
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