BackgroundBefore the start of the project, a basic literature review was conducted to understand the basis of the project, including any existing studies that overlap with my research and the studies directly linked to my invertigation. Muscular dystrophy (MD) is a genetic disease that weakens the muscles that help the body move. People with DM have incorrect or missing information in their genes, which prevents them from producing the proteins essential for healthy muscles. MD is genetic, so people are born with the problem – it's not contagious and you can't catch it from someone who has it. MD weakens muscles, so people with the disease may gradually lose the ability to do most physical activities, such as walking. A person with DM may begin to have muscle problems from birth or later in life. There are over 30 types of DM with various symptoms, but this particular article will explore Duchenne muscular dystrophy (DMD). Duchenne muscular dystrophy is a neuromuscular disease. This is the most common type of MD Duchenne muscular dystrophy. (DMD) is an X-linked disease that affects about 1 in 3,500 men. Women usually carry the defective gene that causes the disease. According to Mendelian inheritance, when a mother carries the defective gene, her daughter will have a 50% chance of being a carrier and her male child will have a 50% chance of having the disease and showing symptoms. . Although the disease is present from conception, symptoms usually appear before the age of 5 or 6. (NHS UK). DMD is the result of a mutation in the dystrophin gene, located on the X chromosome (gene locus Xp21.2). This explains why male offspring are particularly sensitive. DMD does not produce any functional dystrophin protein. The protein, ca...... middle of paper ......electroporation, microbubbles, and ultrasound.The challenges of gene therapyThe common problem with gene therapy is the concern over immune rejection by the target cell. Foreign proteins and DNA are introduced into the body, often impairing an immune response. Even for the use of plasmids, although they do not contain any protein since they are "naked" DNA, the product will always be a protein which, in any case, can induce an immune response from the organism . Another obstacle is that the expression of the desired gene product is often localized in the tissues into which the vectors are introduced. Thus, vectors targeting muscles must be developed to enable systemic treatment of DMD. Finally, the use of viral vectors to introduce genetic material may carry a risk of insertional mutagenesis because a fraction of the viral vector may be integrated into the host genome...