Technology

ASO drugs target disease at the genetic level

An AntiSense Oligonucleotide (ASO) is a short, synthetic piece of DNA-like molecule that can be used to treat a variety of diseases -  by binding to specific messenger RNA (mRNA) strands, which are the blueprints for proteins. ASO drugs can modulate the abnormal protein production associated with specific diseases.  

ASO drugs have shown promise in treating a wide range of diseases:

• Genetic disorders: such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).

• Neurological diseases: like amyotrophic lateral sclerosis (ALS).

• Infectious diseases: including hepatitis B and C.

• Cancer: by targeting specific cancer-related genes.

Advantages:   Specificity,  Potency,  Versatility.

Challenges and future directions

While ASO drugs offer great potential, there are still challenges, such as:

• Delivery: Getting ASOs to the right cells in the body can be difficult.

• Immune response: Some people may develop an immune reaction.
  
  

Research into ASO drugs is rapidly accelerating.

Expect many more ASO based therapies to be developed in the near future.