22 March 2022 | News
In collaboration with DART and AIIMS Jodhpur
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The Indian Institute of Technology (IIT) Jodhpur has established a research centre for Duchenne Muscular Dystrophy (DMD) in collaboration with Dystrophy Annihilation Research Trust (DART), Bengaluru and the All India Institute of Medical Sciences (AIIMS), Jodhpur.
The current therapeutic options available to treat DMD are minimal and highly expensive (over Rs 2-3 crore per child a year). Drugs are mostly imported from abroad, accelerating dosing costs and putting them out of reach for most families.
DMD is a rare and incurable genetic disease and it is the most common and fatal type of muscular dystrophy, marked by gradual muscle deterioration.
The centre at IIT Jodhpur aims to develop affordable therapeutics for DMD and enhance the efficacy of Antisense Oligonucleotide (AON)-based therapeutics.
The AON-based therapeutics' idea is to hide or mask specific exons (a segment of a DNA or RNA molecule containing information coding for a protein) in a gene sequence. In DMD patients, one or more exons can be masked with specific molecules called AON or molecular patches. Due to these challenges, DMD patients need personalised medicine.