Question

Gene therapy attempts to correct a genetic deficiency by inserting a normal version of the affected gene into the patient's cells. For this to be effective, the cells need to be _______cells that will not passed on to offspring, but they also need to be cells that will keep dividing for the lifetime of the patient.  Currently, ______ cells, which produce all the blood and immune cell types of the body, are a preferred target.What limits the use of gene therapy is that it works best on relatively simple conditions caused by the mutation of one single gene.  Retroviruses are ________viruses that insert a(n) ________copy of their genome into that of their host cells.  After the insertion, the viral genome will be permanently part of the host cell genome.

Answer 1

Gene therapy attempts to correct a genetic deficiency by inserting a normal version of the affected gene into the patient's cells. For this to be effective, the cells need to be somatic cells that will not pass on to offspring, but they also need to be cells that will keep dividing for the lifetime of the patient.  Currently, hematopoietic cells, which produce all the blood and immune cell types of the body, are a preferred target. What limits the use of gene therapy is that it works best on relatively simple conditions caused by the mutation of one single gene.  Retroviruses are RNA viruses that insert a (n) single copy of their genome into that of their host cells.  After the insertion, the viral genome will be permanently part of the host cell genome

Gene therapy is an advanced technique that is employed to treat a disease.it is accomplished by inserting a normal gene in the dividing cells of the body. Here somatic cells are discussed, which are body cells that constitute all the cells in tissues organs and blood. Germline cells are those that take part in reproduction. So any change in germ line cells will be inherited to the offspring.

All blood cells and immune cells arise from hematopoietic origin. These are the pluripotent cells that have the ability to differentiate into any type of blood /immune cells.

Retroviruses are group of viruses from retroviridae are RNA viruses. Unlike other viruses retroviruses are diploid in nature. They cannot complete their life cycle and reproduce without integrating their genome in the host cell’s genome; this makes them an efficient tool for gene therapy. Here they are modified and the gene to be corrected is genetically incorporated to the viral genome. When the virus infects the cell one copy of its genome is integrated into host which then replicates normally.