Research Ppaer with draft Paper Example | Topics and Well Written Essays - 750 words - 1

Ppaer with draft - Research Paper Example Since gene mutations primarily cause the condition, attempts to develop gene and cell therapies provide a possible cure for the various types of Muscular Dystrophies. However, gene and cell therapies come with several challenges, especially since the skeletal muscle is the most abundant in the human body. Gene therapy involves gene replacement or modification. As a result, the need to find an efficient method to deliver the new gene to the body becomes of paramount importance. One of the challenges facing gene therapy is the packaging of genes. For example, dystrophin, whose defects are responsible for DMD and Becker MD. Dystrophin is larger than the packaging capacity of many vectors used in delivering the gene to the skeletal muscles. According to Chamberlain 2002, truncated versions of the dystrophin gene become the solution. Research shows that truncating the Central Rod and the C-terminal domains causes minimal changes on the functionality of the dystrophin gene. Shortened versions of the dystrophin gene tested on mice in preclinical studies provide positive results indicating that the micro-dystrophins reverse the abnormalities of the dystrophic muscle.(Cossu & Sampaolesi, 2007) Gene therapy faces the challenge of identifying a favorable viral vector focusing on Adenoviral vectors (Ad), retroviruses and adeno-associated viruses (AAV). Ad vectors contain large capacity of cloning and efficiently infect the muscle. Development of the ‘gutted’ Ad tackles the problem of immune response triggered by the Ad vector. According to Chamberlain (2002), the gutted version contains the ability to package full-length cassettes of dystrophin. However, the Ad vector’s large size hinders diffusion in muscle tissue. Hence, Ad vector is not the best choice vector. Retroviruses posses small cloning capacity and hence are limited to the delivery of mini dystrophins. The most promising gene delivery vector proves to be adeno-associated virus (AAV). (Haidet, Mendell &