Due to the fact that this is still a new scientific breakthrough, the safety concerns outweigh quite a bit of the procedures. Anthony A. Amato, Robert C. Griggs, in Handbook of Clinical Neurology, 2011. List of the Pros of Gene Therapy 1. However, the relatively short duration of protein activity presents practical limitations and, hence, provides a potential need for longer duration protein production at the disease site via gene-based approaches. These therapeutic proteins are often normally expressed in tissue- and/or condition-specific manners. Greely, in International Encyclopedia of the Social & Behavioral Sciences, 2001. The genetic aspects of somatic cell gene therapy have been largely uncontroversial. Gene therapy technique has successfully cured male infertility where DNA is injected into body tissues to replace the abnormal genes causing the sterility. To achieve long term results much more research is needed. D. Neri, in Encyclopedia of Applied Ethics (Second Edition), 2012. Somatic gene therapy pinpoints on the improvement of genetic disease by handling of nonreproductive or somatic tissues. This method of gene therapy includes the exclusion of some of the dysfunctional cells and introducing them with a cloned wild-type gene. Secreted proteins act both on transduced and nontransduced cells, therefore the requirement for a high percentage of cells to be transduced is reduced. A requirement for this form of non-autologous somatic gene therapy is that the recombinant gene product for therapy must be a secretory protein. The social acceptability of risk will then depend on whether people may clearly perceive their own benefits at it. From 2002, the donations have continued to increase annually, reaching a sum of 104 million Euros in 2004 (see Fig. The donations to the “Téléthon” seem to indicate this. Concerning the vector: once is is administered, there is no way for certain to tell if the vector will travel to the intended location. We use cookies to help provide and enhance our service and tailor content and ads. M POTTER, ... P CHANG, in Artificial Cells, Cell Engineering and Therapy, 2007. The first is to pose restrictions on the reproductive activities of persons who have access to SGT or to traditional therapies for genetic conditions, hoping to minimize the disgenic trend. This means it must be under the control of the sequence of DNA that is responsible for switching the gene on. Instead of paying for hospice care or being forced to say goodbye immediately, doctors and scientists a… Here is where you can find all of these things concerning Gene Therapy. 1. The equivalence is strengthened by the fact that the introduced modification (which may be corrective, but also ameliorative) affects only the individual subjected to medical treatment and is not passed on to progeny. The technique of somatic gene therapy involves incorporating a normal gene into the suitable cells of an individual affected with a genetic disease, thereby permanently correcting the disorder. The second hurdle is insertion of gene into the right cells. Many of the children born in this demographic die soon after birth because of the devastating effects of their condition. Shailendra Dwivedi, ... Kamlesh K. Pant, in Omics Technologies and Bio-Engineering, 2018. It was initially conceived as introducing a properly functioning copy of a gene into a person who had a genetic disease as a result of inheriting only improperly functioning copies. The final obstacle is making sure the gene is active. There are several factors that make it unlikely that the initial successes in gene therapy will occur through replacement of a missing or defective chromosomal gene (the exception of genes encoding secreted proteins is discussed below): (1) the current gene transfer technologies may not be efficient enough to transduce a sufficiently high percentage of affected cells in a single tissue to restore function, (2) many diseases affect multiple organs and may require widespread gene transfer throughout the patient's body, (3) it is unclear whether gene replacement will reverse long-established symptoms presented by the patient, and (4) the technologies for transferring large segments of DNA capable of bearing appropriately regulated promoters are only beginning to be developed.
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