Gene Literature Review: Germline Gene Therapy

Gene therapy is a technique that uses genes (sections of DNA) to treat or prevent a disease.

All cells within an organism’s body contain genes making them potential targets for gene therapy.

(Each molecule of DNA is made up of two strands twisted around each other. Each strand is constituted of four ‘nucleotide bases’, called A, T, G, C, and these bases on the two strands, pair in an A-T, G-C formation.) DNA designs life; proteins express it. Even though DNA is correctly translated into proteins 99.999 % of the time, just one wrong chemical base in one gene of one chromosome can declare a child to a wheel chair, or lead to cystic fibrosis or muscular dystrophy. The only solution to this issue is to fix those genes. Finding a supplement gene

Germline cells are reproductive cells such as; sperm or eggs. You can fix somatic cells via somatic gene therapy, and cure the patient. But the patient will still carry the same genetic abnormalities in the genes of their Germline cells. The result from this is that the patient’s child may still inherit that genetic disease. On the other hand with Germline gene therapy, you cure the child before it is born and that child’s descendants.

May 14, 2000 (Boston) – Although some people think gene therapy is “monkeying around with nature”, researches in Ohio and California have shown that it may be possible to correct some genetic diseases in babies while they are still in their mothers womb. The gene transfer technique was discussed at a joint meeting of the pediatric Academic Societies and the American Academy of pediatric uses harmless viruses to carry copies of a normal gene (building blocks of a person) to foetuses with missing or defective copies of gene. Once in the body, the transferred gene can direct the foetuses blood and bone marrow cells to swirl out the proper protein needed to prevent damage to cells and tissues that would otherwise

Somatic gene therapy only involves the insertion of therapeutic DNA into body cells and not the germ cells or gametes. That means that any effects that occur are confined to that individual being treated and are not inherited by future offspring.

During human fetal development, there is an opportunity that exists for the therapeutic insertion of genes without incurring the displeasure of the immune system. The foetus is an immune tolerant site( doesn’t have the ability to mount a response to the antigen) that is full of progenitor cells ( a cell that originates from a stem cell and differentiates into a more specialised cell ) designed to last a life time. The insertion of genes into this responsive environment could provide the opportunity to correct gene defects and prevent diseases, instead of trying to treat their destructive effects. In uterus gene therapy, it offers several advantages in the treatment of genetic disorders. The large amount of somatic stem cells readily available for gene transfer in the foetus has made this a promising technology. The permanent replacement of a gene into somatic stem cell would ensure that daughter cells would also carry the gene, eliminating the need of repeated therapy during the lifetime of the affected individual. Researches have suggested though that it is difficult to search