Exclusive | Can gene editing and oligonucleotides reverse the development process of Huntington's disease?

▎Editor's note from WuXi AppTec's content team: According to estimates by the World Health Organization, there are currently 6000-8000 rare diseases in the world, affecting the lives of up to 400 million patients.

Rare diseases are closely related to everyone.
As long as there is a life inheritance, there is a possibility of rare diseases.

With the efforts of various parties, rare disease drugs have gradually become an important part of the development of innovative drugs.

Huntington's Disease (Huntington's Disease) is a rare disease that is inherited from a single gene and affects the nervous system.

The patient's gene duplicates and amplifies a DNA sequence, resulting in the production of abnormal proteins.

At present, only symptom-relieving drugs are on the market, and there is still no therapy to control the progression of the disease.

With the advancement of research in the field of life sciences, therapies spawned by new strategies are no longer limited to conventional small molecules.

This analysis series focuses on oligonucleotide therapy and gene editing therapy.
Based on scientific papers and patent activity data, some laws and logic behind the data have been discovered.

I hope that this will contribute to the research and development of new drugs in this field, help rare disease groups including Huntington's disease, and jointly take care of our future.

If you have any questions, please contact data_news@wuxiapptec.
com.

We look forward to your feedback and suggestions! Note: This article aims to introduce the progress of medical and health research, not to recommend treatment options.

If you need guidance on treatment plans, please go to a regular hospital for treatment.