Approach
Scientists have taken the logical step of trying to introduce genes directly into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy and sickle cell anemia. However, this has proven more difficult than modifying bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering them to the correct site on the gene. Today, most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. Antisense therapy is not strictly a form of gene therapy, but is a related, genetically-mediated therapy.
The most common form of genetic engineering involves the insertion of a functional gene at an unspecified location in the host genome.This is accomplished by isolating and copying the gene of interest, generating a construct containing all the genetic elements for correct expression, and then inserting this construct into a random location in the host organism. Other forms of genetic engineering include gene targeting and knocking out specific genes via engineered nucleases such as zinc finger nucleases, engineered I-CreI homing endonucleases, or nucleases generated from TAL effectors. An example of gene-knockout mediated gene therapy is the knockout of the human CCR5 gene in T-cells in order to control HIV infection. This approach is currently being used in several human clinical trials.
Read more about this topic: Gene Therapy
Famous quotes containing the word approach:
“You should approach Joyce’s Ulysses as the illiterate Baptist preacher approaches the Old Testament: with faith.”
—William Faulkner (1897–1962)
“This is an approach to that universal language which men have sought in vain.”
—Henry David Thoreau (1817–1862)
“The modern world needs people with a complex identity who are intellectually autonomous and prepared to cope with uncertainty; who are able to tolerate ambiguity and not be driven by fear into a rigid, single-solution approach to problems, who are rational, foresightful and who look for facts; who can draw inferences and can control their behavior in the light of foreseen consequences, who are altruistic and enjoy doing for others, and who understand social forces and trends.”
—Robert Havighurst (20th century)