Navigation Links
Gene Therapy For Bubble Baby Syndrome May Lead To Blood Cancer

Gene transplant, a technique to treat the so-called bubble babies, afflicted with a genetic disorder called Severe Combined Immunodeficiency (SCID), may actually be causing more harm// to the patient than thought. It is now suggested that the gene transplant procedure activates a cancer-causing gene, predisposing the patient to leukemia and death.

Reports of leukemia related death of a young boy as a consequence of gene therapy trial treatment of SCID has raised concerns about the use of this technique as a cure for this serious hereditary immune disorder.

Mutation in a gene called IL2RG is known to cause SCID. The protein product of this gene plays a crucial role in the growth of immune cells. Any abnormality in this protein leads to an increased susceptibility to infections. Children suffering from this disease therefore have to protected in a sterile environment (a bubble like encasement) and hence the name.

Gene therapy or rather gene transplant involves introduction of the corrected version of the gene (normal gene) into the body. A study conducted on mice at the Salk Institute in California under the guidance of Prof Inder Verma highlights that this gene replacement can trigger cancer. The result of this controveraisl study is published in the most prestigious Nature journal.

Nearly one third of the mice treated with the normal version of the IL2RG gene developed a form of blood cancer. 'Curing X-SCID by replacing IL2RG in the manner it is currently being done puts patients at an increased risk of developing cancer,' said Dr Niels-Bjarne Woods, a researcher involved in the gene therapy trial.

However, Prof Adrian Thrasher, has something different to say. 'On the basis of the data that is published, which is very preliminary, it is quite possible that the effects are caused by the artificially high levels of the gene in the animals. Certainly we and our colleagues have found no evidence for this effect,' he remarked.

Prof Adrian Thrasher has treated eight boys suffering from this life-threatening disorder with the normal gene at the Great Ormond Street Hospital. Additionally, he has treated two young victims afflicted with Chronic Granulomatous Disorder (CGD) using gene transplant, both of whom managed to clear infections that had not responded to treatment previously.

However it is not known about the long-term side effects associated with form of treatment. Concerns of the scientific community on one side, nothing seems to deter the hope of patients like Mark, a 6 year old CGD sufferer, who feel that gene therapy offers a ray of endless hope at the end of the tunnel.

Clearly, more studies are indicated to accurately assess the risk and benefit ratio associated with gene therapy before it can be commercialized.


'"/>




Related medicine news :

1. Cancer Doctors Okays Controversial Prostate Therapy
2. Consensus on "Combination Therapy" for Breast Cancer
3. Gene Therapy shows promise in treating Hemophilia
4. "Make AIDS Therapy affordable" - Physicians demand
5. Hormone replacement Therapy a headache
6. Simple Therapy
7. Therapy for stopping the spread of cancer cells
8. Gene Therapy Destroys Pancreatic Cancer Cells
9. Letrozole Beats Tamoxifen in Breast Cancer Therapy
10. Garlic Supplements Impede HIV Therapy
11. Gene Therapy For Cystic Fibrosis
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:2/5/2016)... , ... February 05, 2016 , ... ... setting the stage for new clinical and scientific initiatives have all marked the ... she was appointed President and CEO of the nation’s oldest cancer center, Candace ...
(Date:2/5/2016)... (PRWEB) , ... February 05, 2016 , ... ... College President George H. Van Allen have signed a joint enrollment and degree ... a seamless pathway toward associate and baccalaureate degrees at FHU|Dickson. , The ...
(Date:2/5/2016)... ... 05, 2016 , ... The American public tends to feel uncomfortable about drinking ... regular municipal or well water. The recent experience with lead contaminated water in Flint, ... long way toward increasing public acceptance of recycled waste water as drinking water. ...
(Date:2/5/2016)... ... 05, 2016 , ... At its annual meeting held last week, the American ... National Board of Directors. Mr. McDermott succeeds former APDA Chairman, Fred Greene. , "We ... Chambers , APDA President and CEO. “Pat has tirelessly served APDA since 2001 when ...
(Date:2/5/2016)... ... February 05, 2016 , ... In sleep, when the defenses of the day ... feature of patients with eating disorders is significant self-criticism, and consequently these patients experience ... are regarded as maladaptive means for coping with this unease, but also leads to ...
Breaking Medicine News(10 mins):
(Date:2/5/2016)... CENTENNIAL, Colo. , Feb. 5, 2016  As ... about health. The multitude of recommended screenings and tests ... healthy aging a priority. However, for the majority of ... of proactive health planning. For the 37.5 million American ... time like the present to make hearing health a ...
(Date:2/5/2016)... 2016 Aethlon Medical, Inc. (Nasdaq: AEMD ), ... will be presenting at Source Capital Group,s 2016 Disruptive Growth ... at 2:15 p.m. ET on Wednesday, February 10, 2016.  ... taking place at 3:15 p.m. ET. http://www.aethlonmedical.com .  ... after the conclusion of the live event. The panel discussion ...
(Date:2/5/2016)... 2016  Zimmer Biomet Holdings, Inc. (NYSE and SIX: ... underwritten secondary offering of 11,027,558 shares of its common ... of Blackstone and Goldman Sachs.  The shares are being ... $96.45 per share. The selling stockholders will receive all ... nor any of its directors, officers or other stockholders ...
Breaking Medicine Technology: