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Transcript
Research Review No. 91. An interesting review. The Studies: In the latest issue of Frontiers in Bioscience-Landmark, there is a very useful and comprehensive review of the genetic therapies for Duchenne Muscular Dystrophy (DMD), which are published in the world literature. The three French researchers who co-authored this review are experts in this field of endeavour and therefore their views on the various treatments for neuromuscular disorders (NMD) are very valuable. They state at the opening that: “In this review, we will focus on the main advances in gene therapy and RNA-based approaches for the treatment of NMDs. In their review they concentrate on DMD and Spinal muscular atrophy (SMA), but I will restrict myself to DMD. While there is currently no effective therapy to prevent or affect the gradual muscle loss associated with DMD they consider that the studies, which “include gene therapy aiming at reintroducing a functional recombinant version of the mutated gene using adeno-associated, lentiviral or adenoviral vectors, as well as RNA-based strategies using antisense oligonucleotides (AONs)”. They consider that these hold great promise for the future and they discuss the clinical trials currently in progress especially for DMD. They discuss and describe the studies currently in progress in which “a DNA coding sequence (cDNA) from a native gene, constructed and designed to be transcribed and then translated into a protein to compensate the deleterious effect of the mutated form directly or indirectly responsible for the genetic disorder.” However a present the problems of introducing the full gene for dystrophin is raising problems. Nevertheless these problems are not considered unsurmountable. Various viral vectors and other modes of introduction of the gene for dystrophin are in progress and showing promise. There is also progress reported in overcoming the immune responses to the introduction of a ‘foreign’ virus into the human body. Exon skipping has been discussed for many years since it was first demonstrated in 1996 and “Since then, numerous in vivo studies have provided pre-clinical evidence for the therapeutic potential of an antisense strategy for DMD in several animal models”. The authors follow their discussion by stating that: “Following the encouraging results obtained on these animal models, antisense mediated exon skipping has rapidly moved towards clinical evaluation in DMD patients.” They proceed to describe these investigations in detail. The authors’ final conclusions sound very positive and I quote their final sentence in full: “With continued success, we will hopefully see a gradual implementation of new therapies over the coming years that will increasingly extend lifespan and improve the quality of life for patients with neuromuscular diseases”. Reference: 1) Benchaouir, R., Robin, V. & Goyenvalle, A. (2015) Gene and splicing therapies for neuromuscular diseases. Frontiers in BioscienceLandmark. 20:1190-1233. Karl A. Bettelheim 8.6.2015