Exploring the Potential of Gene Therapy Applications in Duchenne Muscular Dystrophy Patients through the Development of a Construct Containing Telomeres, Centromere, and Origins of Replication
Duchenne muscular dystrophy (DMD) is a severe, progressive form of muscular dystrophy caused by mutations in the dystrophin gene (Kadkhodaei et al., 2021). While there are currently no approved treatments for DMD, gene therapy holds potential for therapeutic interventions. To this end, the development of a construct containing telomeres, a centromere, and origins of replication would allow for the encoding of the entire DMD gene and its regulatory sequences (Gueye et al., 2016). The inclusion of telomeres, which are specialized DNA sequences that act as a protective cap at the ends of chromosomes, would ensure the stability of the construct once it is integrated into the host genome. The centromere, which is a region of DNA that is essential for the proper segregation of chromosomes during cell division, would allow for the expression of the construct in a number of cells. Finally, the inclusion of origins of replication, which are regions of DNA that allow for the replication of the construct, would facilitate the efficient expression of the construct in the target cells. Cont…
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