All About Somatic Gene Therapy
Somatic gene therapy refers to the specific gene therapy that involves insertion of new genetic material into body cells and tissues other than the reproductive ones.
As a result of this therapy, the new cells introduced into the body are not passed on to the next generation because they are not part of the sperm cells or egg cells but only of the body cells.
This kind of gene therapy changes the genetic coding of specific cells of a human body by effective means of DNA delivery system. The genetic alteration is also, at times, done using two methods namely In-Vivo or Ex-Vivo therapy. The uses of this gene therapy are being experimented and developed at a fast-paced rate. At present, medical professionals are making use of this therapy to cure severe combined immune deficiency, muscular dystrophy, hemophilia, and many other genetic disorders and acquired diseases.
What is Germ Line Gene Therapy
In a regular gene therapy, defective genes are replaced or altered in the body cells of a organism. In such a case, only the organism receiving the treatment gets the benefit and treatment from a particular disease. Germ line gene therapy is a specific kind of therapy where genes are inserted into the reproductive cells/tissues of the organism.
It is an approach where in the fresh and healthy genes are delivered to egg cells or sperm cells. This kind of a therapy reduces the risk of defective genes being transferred to the next generation of the organism.
Germ line gene therapy can be used in two ways. The first method involves the alteration of the genetic makeup of reproductive tissues before they are fertilized. The second method involves altering the genetic makeup of blastomere at its early stage. The target of this therapy is to change the genetic code of an organism and pass it on to the subsequent generation. In the former case, the mutations are transferred to future generation. And in the latter, the altered cells are permitted to go on dividing and then developing into animals. Some of these cells will possess the genetic alteration and others will not.
Risks of Gene Therapy
There are a number of risks associated with gene therapy. In the beginning phases, there can be problems that result from the way the genes are delivered.
Because delivery of the gene into the cells by a direct method is not an easy process it usually requires delivery through something known as a vector which is another word for a carrier. Viruses are the most common types of gene therapy vectors because they can identify certain cells and transport genetic material to genes within the cells. There is research currently being conducted which calls for removing the genes that cause disease from the viruses and replacing them with a particular gene that is needed to end the disease. The process next calls for the altered viruses to be inserted into the diseased cells of a person where their genetic material can be delivered. There are risks associated with this process such as:
Recent HIV Treatment Developments In Gene Therapy
There have been a lot of discoveries in the last few years regarding gene therapy. A variety of diseases and genetic malfunctions in the human body are being addressed through these treatments. Three of the most recent among them offer hope for HIV sufferers. The developments in gene therapy push the boundaries of medical science further, every day.
There are diseases and genetic orders of the past which have been readily treated with this sort of therapy despite former beliefs that no treatment might be possible.
Antiretroviral drugs designed to treat HIV have shown remarkable progress. However, they do not cure the affliction and the CD4 (white blood cell) count will usually remain quite low after using these drugs. Gene therapy has been discussed and beginning tests have shown positive results.
What Diseases Can be Treated with Gene Therapy
In simple words, gene therapy refers to the procedure where specific genes are inserted or altered into the cells and biological tissues of a human body to correct the defected ones. It directly involves correcting the mutation and modification of normal genes, which can cause a viral infection. A number of success stories of this therapy have come to the forefront.
This therapy has been and can be used for the treatment of many diseases. Read on to find out what diseases can be treated with gene therapy.
Genetic Disorders ? It is used for the cure for the following genetic disorders:
? Severe Combined Immune Deficiency (ADA-SCID) ? Also known as the bubble boy diseases, under this disease a child is born with a very weal immune system. Bone marrow transplantation is required. Through this therapy, ADA is inserted and then the genetically corrected cells are transplanted back into the patient?s body.
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Gene Therapy In The News
- $5 million gift from Miami couple to boost gene therapy research at the ... - University of Florida
- apceth and Indivumed to Develop Biomarker Platform for Targeted Cellular Therapies - MarketWatch (press release)
- E'shee Clinical Esthetic Launches High-Tech Skin Serum - Bradenton Herald
- CHMP asks AMT to provide additional information on Glybera - pharmabiz.com
- E'shee Clinical Esthetic announce Elixir of Life KI Therapy Serum - eNewsChannels
- Gene therapy could prevent seizures, study finds - Epilepsy Research UK
- Gene therapy helps regenerate injured brain cells - Times of India
- Gene therapy for epilepsy could stop seizures, UF researchers say - University of Florida
- Scientists Use Gene Therapy to Restore Eyesight - Voice of America
- Gene Therapy for Inherited Blindness Succeeds in Patients' Other Eye - Sacramento Bee

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