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:10/13/2017)... ... ... While it’s often important to take certain medications during the night, finding ... identified a solution. , She developed a prototype for MOTION LIGHT-UP PILL BOX to ... the need to turn on a light when taking medication during the night, allowing ...
(Date:10/13/2017)... ... October 13, 2017 , ... PurhealthRX , a leading Health and Nutrition ... the Purzorb™process to full spectrum CBD oil will revolutionize the rapidly growing CBD market ... can be easily incorporated into liquid products, while reducing costs to end users. , ...
(Date:10/13/2017)... Shelton, CT (PRWEB) , ... October 13, 2017 , ... ... and long-term care services, staged a mock evacuation of the facility as part of ... Department, Shelton Fire Department, Echo Hose EMS and Shelton City Emergency Manager, as well ...
(Date:10/13/2017)... ... ... Ellevate Network, the leading network for professional women, brought together some of ... their inaugural Summit in New York City in June. The event was livestreamed with ... 3 million. To watch the Mobilize Women video, click here . , ...
(Date:10/13/2017)... ... ... Talented host, actor Rob Lowe, is introducing a segment ... of "Success Files," which is an award-winning educational program broadcasted on PBS Member ... with passion and integrity. , Sciatica occurs when the sciatic nerve in the ...
Breaking Medicine News(10 mins):
(Date:9/28/2017)... Sept. 28, 2017 Hill-Rom Holdings, Inc. (NYSE: ... conference call and webcast on Friday, November 3, 2017, ... and ending at approximately 8:30 a.m. (CDT) / 9:30 ... company,s 2017 financial performance and guidance for 2018, Hill-Rom ... to enhance operational performance, and long-range financial outlook through ...
(Date:9/25/2017)... Sept. 25, 2017  EpiVax, Inc., a leader ... and immune-engineering today announced the launch of EpiVax ... of personalized therapeutic cancer vaccines. EpiVax has provided ... access to enabling technologies to the new precision ... lead EpiVax Oncology as Chief Executive Officer. Gad ...
(Date:9/19/2017)... , Sept. 19, 2017   ZirMed Inc ., a ... today announced that it has been ranked #1 by its ... Book™ Rankings 2017 User Survey. ZirMed was recognized as ... large hospitals and medical centers over 200 beds and holds ... healthcare technology user survey history. ...
Breaking Medicine Technology: