Research Index


1. Exon skipping using antisense oligonucleotides Exon skipping employs synthetic DNA-like molecules called antisense as a "DNA band-aid" to skip over the parts of the gene (exons) that block the effective creation of dystrophin.More info
2. Stop codon read through Around 15% of DMD patients have stop codon mutations. Some experimental molecules can cause muscle cells to "read through" or “ignore” erroneous stop signals in the gene.More info
3. Exon skipping with gene transfer Exon skipping can also be induced by recombinant virus vectors containing modified small nuclear [sn] RNA gene.More info
4. Gene therapies Gene therapies typically employ recombinant virus vectors and aim to correct or induce genes to rescue the pathology of DMD.More info
5. Cell therapies Various types of stem cells give rise to muscle progenitor cells and potentially rescue muscular dystrophies.More info
6. Utrophin Upregulation Utrophin is a protein similar to dystrophin and compensate for loss of strength and function due to defective dystrophin in muscles.More info
7. Myostatin inhibition Myostatin inhibition leads to muscle hypertrophy and potentially ameliorate the pathology of muscular dystrophies.More info
8. TGFβ Inhibition/Anti-Fibrosis Various experimental molecules can block TGFβ-mediated collagen synthesis. More info
9. NF-kB inhibition/anti inflammatory Several experimental molecules reduces the adverse effects of NF-κB signaling.More info
10. TNF-alpha inhibition TNF-alpha is pro-inflammatory, pro-fibrotic, and pro-NFKB. TNF-α is found to be elevated in DMD and in mdx muscles. More info
11. Anti-Oxidants Anti-oxidants decrease muscle necrosis in dystrophic mdx mice and protects against reactive oxygen species. More info
12. IGF-1 A growth factor IGF-1 is involved in muscle hypertrophy.More info
13. α7-integrin upregulation Increasing α7-integrin promotes muscle cell proliferation, adhesion, and resistance to apoptosis.More info
14. nNOS pathway enhancement nNOS is a member of dystrophin complex and involved in vasodilatation.More info
15. Angiotensin converting enzyme (ACE) inhibitor/ beta-blocker for cardiomyopathy management Angiotensin-converting enzyme inhibitors are used in the management of cardiomyopathy. More info
16. Corticosteroid Corticosteroid is a useful palliative treatment.More info
17. Other Other miscellaneous strategies.More info