AP BIOLOGY

Detailed explanation-1: -Gene therapy is used to treat genetic disorders resulting due to the presence of a nonfunctional gene copy. In this case, functional genes are inserted into the cells or tissue of a patient and then those cells are delivered to the patient to compensate for the effects of the non-functional gene.

Detailed explanation-2: -When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient. When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab.

Detailed explanation-3: -Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms: Replacing a disease-causing gene with a healthy copy of the gene. Inactivating a disease-causing gene that is not functioning properly.

Detailed explanation-4: -Gene therapy has some potential risks. A gene can’t easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector. The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells’ genes.

Detailed explanation-5: -Usually, insertions and the subsequent frameshift mutation will cause the active translation of the gene to encounter a premature stop codon, resulting in an end to translation and the production of a truncated protein.