Use of anti-miR106b inhibitors for promoting muscle regeneration in the treatment of Duchenne muscular dystrophy (DMD)
University of Jaén
Non-confidential report
The research group led by Dr Aránega proposed a novel approach – the use of miR-106b inhibitors as a potential therapy to increase the regenerative capacity of the dystrophic satellite cells in Duchenne muscular dystrophy (DMD). If successful, this treatment would not be dependent on the type of mutation in DMD and could potentially be used for other muscle wasting conditions.
However, this was a very early application and the panel felt that a suitable lead compound needed to be identified before advice could be given on the development of a clinical trial. The panel posed questions at a pre-clinical level with recommendations that experiments use the standard operating procedures available from the TREAT-NMD website. The advice also recommended further pre-clinical work including the replication of data in an independent lab with a robust power analysis informing the optimal number of animals that should be used.
Name of applicant: Amelia Aranega
Reviewed: November, 2018 in Chicago, IL, USA
The research group led by Dr Aránega proposed a novel approach – the use of miR-106b inhibitors as a potential therapy to increase the regenerative capacity of the dystrophic satellite cells in Duchenne muscular dystrophy (DMD). If successful, this treatment would not be dependent on the type of mutation in DMD and could potentially be used for other muscle wasting conditions.
However, this was a very early application and the panel felt that a suitable lead compound needed to be identified before advice could be given on the development of a clinical trial. The panel posed questions at a pre-clinical level with recommendations that experiments use the standard operating procedures available from the TREAT-NMD website. The advice also recommended further pre-clinical work including the replication of data in an independent lab with a robust power analysis informing the optimal number of animals that should be used.