Navigation Links
Treatment of Sickle Cell Anemia Using Gene Therapy.

A research was conducted by Selda Samakoglu and colleagues at the Memorial Sloan-Kettering Cancer Center. This research opened new doors in the treatment of the genetic disease, Sickle cell anemia // . The treatment combines both gene therapy and RNA interference therapy. So far the treatment has worked well in the laboratory stage and will be of great success in curing the disorder.

Sickle cell disease is a genetic disease affecting 80,000 people in the US alone. The Red Blood Cells (RBCs) become lose there normal disk shape and become sickle shaped. Due to this abnormality there is hindrance in the movement of these sickled RBC. The movement inside the small blood vessels is restricted and this results in piling, clumping inside the vessels. This eventually blocks the vessels leading to stroke, pain, infection and organ damage.

This disease is very much prevalent among the Africans, Mediterranean, Indian or Middle Eastern individuals. At present the treatment is that the affected people are given a stem cell transplant from a donor. It is very difficult to find an exact mach and less than 20% of the children suffering from the diseases find a complete donor match.

The researchers said that at the moment they are working with animal model (mice) and after the tests results prove to be positive they will start human trails and bring about the treatment procedure in the main stream of medicine within a few years.

The stem cells taken from the bone marrow are cultured in the lab and a small RNA sequence that interferes with the production of beta S-globin is integrated. This is because this sequence that produces beta S-globin is altered in these patients hence the altered gene is prevented from getting translated to proteins. This set up is then inserted in a gene that promotes production of healthy hemoglobin chains.

The net result is the production of a transgene which is then inserted into a vector (virus) and i s introduced into cell cultures. This virus is then taken into the stem cells of the sickle cell patients. After that the cells don’t produce beta S-globin but functions like the normal healthy Red Blood Cells. Further studies will be conducted even on the genetic disease Thalassemia which will be very useful in conducting human trials and studying the results of their sickle cell therapy.

The medical community appreciated the research and said the faster the cure more the lives will be saved. At present 1000 children are suffering from the disease and it would save their lives.

But they also warned that genetic therapy has its own disadvantages and complications similar to the various gene therapies. All of it has to be foreseen and eliminated for a safe procedure without any side effects to tackle sickle cell anemia.


'"/>




Related medicine news :

1. Advances in Treatment of Cataracts
2. Recommendations for Treatment of Blood Pressue
3. Treatment for Menieres disease
4. Treatment for pre-menstrual syndrome is ineffective
5. Better Treatment for obesity
6. Gene Treatment for Heart Disease
7. More People Seeking Treatment for Depression
8. Focused Treatment For Childhood Cancer
9. Inadequate Drug Treatment For Youth
10. FDA Approves New surgery Treatment for Farsightedness
11. Treatment of antibiotics ineffectual in bronchitis
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:10/13/2017)... (PRWEB) , ... October 13, 2017 , ... PurhealthRX ... CBD Oil utilizing Purzorb™ technology. Applying the Purzorb™process to full spectrum CBD oil will ... and providing a CBD form that can be easily incorporated into liquid products, while ...
(Date:10/13/2017)... ... , ... The Visiting Nurse Association (VNA) of Somerset Hills is proud to ... unique items from across the nation, this holiday-themed event will raise funds and awareness ... VNA. The boutique will be open Saturday, November 4 (10:00 a.m. – 5:00 ...
(Date:10/13/2017)... ... October 13, 2017 , ... ... Software Development, has been awarded a contract by the Center for Medicare and ... aims to accelerate the enterprise use of Agile methodologies in a consistent and ...
(Date:10/12/2017)... , ... October 12, 2017 , ... Planet Fitness, one ... U.S., announced today its plans to open a flagship location in Covington, LA at ... former Rooms To Go store next to Office Depot in the Holiday Square shopping ...
(Date:10/12/2017)... , ... October 12, 2017 , ... ... delivery system that we intend to develop to enable prevention of a major ... to severe hearing loss, especially in pediatric patients. For cisplatin, hearing loss is ...
Breaking Medicine News(10 mins):
(Date:10/4/2017)... Mass. , Oct. 4, 2017  According to the Centers for ... the end of October . PhysicianOne Urgent Care is helping communities across ... Westchester, NY , by offering no-cost* flu shots through the ... as mandated by certain health insurance regulations. ... The best time to get a flu shot is by the ...
(Date:10/2/2017)...  AllianceRx Walgreens Prime, the combined central specialty pharmacy ... benefit manager Prime Therapeutics LLC (Prime), today officially began ... unveiling of new signage at its headquarters in ... a few other company-owned facilities across the country. This ... of whom will begin to see the AllianceRx Walgreens ...
(Date:9/28/2017)... Cohen Veterans Bioscience and Early Signal Foundation announce a ... sensors for real-time monitoring of patients with trauma-related and ... focused on disruptive health solutions for rare disorders and ... record and integrate behavioral, cognitive, physiological and contextual data. ... ...
Breaking Medicine Technology: