التفاصيل البيبلوغرافية
| العنوان: |
P.121 Dnm2 reduction combined with dystrophin re-expression ameliorates the myopathic phenotype observed in the D2-mdx model of Duchenne muscular dystrophy. |
| المؤلفون: |
Menuet, A.1 (AUTHOR), Buono, S.1 (AUTHOR), Robé, A.1 (AUTHOR), Chhor, S.1 (AUTHOR), Eyler, L.1 (AUTHOR), Becker, J.1 (AUTHOR), Colombo, S.1 (AUTHOR), Cowling, B.1 (AUTHOR) |
| المصدر: |
Neuromuscular Disorders. 2022 Supplement 1, Vol. 32, pS99-S99. 1p. |
| مصطلحات موضوعية: |
*DUCHENNE muscular dystrophy, *DYSTROPHIN, *FACIOSCAPULOHUMERAL muscular dystrophy, *MUSCLE strength, *MUSCULAR dystrophy, *MUSCLE mass |
| مستخلص: |
Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy in children. Extensive therapeutic development is ongoing for DMD, with many molecules aiming to increase dystrophin expression, leading to the approval of several exon-skipping antisense oligonucleotide (ASO)-mediated therapies leading to expression of truncated dystrophin. However, to date, no transformative therapy has been approved for this devastating disease. Our preclinical data showed that reducing Dnm2 expression has therapeutic potential in DMD. ASO-mediated Dnm2 reduction resulted in improved muscle strength, as identified by hanging and grip tests, in D2-mdx mice. Increased muscle strength was associated with an increase in muscle mass, and improved muscle histology. Both muscle mass and force improvements were similar to those observed following ASO-mediated re-expression of dystrophin. The strongest improvement in muscle strength was observed following combined Dnm2 reduction and dystrophin re-expression. Given the unmet need with the current therapies available or in development for DMD, combination therapies with the ability to further improve muscle structure and function are much needed. The combination of Dnm2 reduction and dystrophin re-expression may therefore represent a therapeutic avenue, warranting further investigation. [ABSTRACT FROM AUTHOR] |
| قاعدة البيانات: |
Academic Search Index |
