ABSTRACT

This chapter discusses methodology related to changing the expression of a gene. Deoxyribonucleic acid (DNA) and Ribonucleic acid may be administered to patients to modify the expression of a gene. A number of patients with a genetic disorder have been successfully cured with bone marrow or cord blood transplantation. Long terminal repeat (LTR) regions present in the vector contain promoter or enhancer elements that tend to drive the expression of a host gene located next to the LTR region. As an alternative to retroviruses, adeno-associated viruses have been used in gene therapy. Many Duchenne muscle dystrophy (DMD) patients have mutations that cause a shift of reading frame during translation. The chapter examines a specific example of how clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 are used to correct a genomic error. In experiment, DMD patient myoblasts grown in culture were genetically altered with CRISPR/Cas9 to introduce mutations.