ProHealth health Vitamin and Natural Supplement Store and Health
Log In  |  My Account  |  View Cart  View Your ProHealth Vitamin and Supplement Shopping Cart
800-366-6056  |  Contact Us  |  Help

|
|
|
|

Trending News

Missing protein explains link between obesity, diabetes

Fish oil use associated with brain volume preservation

The Brain Boosting and Fatigue Fighting B-12

Genetic basis for distinct type of autism uncovered

Higher vitamin D levels associated with better cancer prognosis

Genetic study suggests causal link between vitamin D deficiency and hypertension

Unsuspected aspect of immune regulation revealed: Role of B cells

IBS, Crohn’s Disease, Colitis, and Other Digestive Disorders

Explaining 'Healthy' Obesity

Sinus Congestion and Sinusitis, Healing Naturally

 
Print Page
Email Article

Scientists Use Gene Therapy to Cure Immune Deficient Child

  [ 10 votes ]   [ Discuss This Article ]
www.ProHealth.com • July 10, 2002




By Judy Siegel-Itzkovich, Jerusalem

An international team of scientists has managed to cure a child with severe combined immunodeficiency (SCID) using gene therapy.

Patients with SCID are usually treated by replacing an essential enzyme, adenosine deaminase, which is lacking in such people. But this treatment is effective for only a couple of days at a time and is expensive. Some patients can have a bone marrow transplant, but only if a good match can be found.

The team was headed by Professor Shimon Slavin, of the Hadassah University Hospital in Jerusalem, and his colleagues Dr Shoshana Morecki and Dr Memet Aker. They collaborated with Dr Allesandro Aiuti and other staff from the San Raffaele Institute for Gene Therapy in Milan, Italy. They published their results in the journal Science last week (2002;296:2410-3).

The patient, whose first name is Salsabil, is a baby Arab girl from east Jerusalem who had to live for the first seven months of her life inside a plastic bubble to protect her from all pathogens because she totally lacked an immune system.

Although the condition is diagnosed in only a few dozen infants around the world each year (in most cases the babies die before a diagnosis is made and they can be treated), Professor Slavin said the treatment can be used to cure other genetic diseases in which patients are born lacking a vital enzyme.

"Among these are Gaucher's syndrome, metachromatic leukodystrophy, and Hurler's syndrome," he said.

A decade ago scientists began treating SCID by using gene therapy to alter T cells and stem cells. Although a theoretical success, these experiments failed to have a real impact on the babies' condition because only a tiny amount of the genetically abnormal bone marrow products was repaired, Professor Slavin explained.

The patients still required adenosine deaminase replacement treatment to survive because the "good" cells were overwhelmed by the much larger number of genetically abnormal cells remaining in the host.

Professor Slavin believed that if he could give the genetically treated cells a "biological advantage," they could overcome the more numerous abnormal cells. He asked Dr Claudio Bordigan, head of the San Raffaele Institute, to supply adenosine deaminase retroviral vector.

At Hadassah University Hospital 15 months ago, Professor Slavin's team gave Salsabil a mild form of chemotherapy, called non-myeloblative conditioning, to suppress her defective bone marrow cells, and prepare the ground for the transfused cells to multiply. The team then introduced, using a genetically engineered virus, a healthy copy of the missing gene for adenosine deaminase into her purified bone marrow stem cells.

No enzyme replacement had been given at any stage, showing that the gene alteration treatment was responsible for the cure.

The baby recovered quickly, and within a few weeks the number of lymphocytes in her blood rose dramatically. Within a month she was out of isolation and went home perfectly well.

She is the third baby with SCID born to her parents. The first child died from the condition and the second, Salsabil's older sister Tasmin, survived after receiving a transplantation of allogeneic umbilical cord blood from a younger brother.

After the first child died, Hadassah doctors froze umbilical cord blood cells from each successive baby for possible treatment of subsequent siblings.

Salsabil's immune system is thought to be working properly, because after she was exposed to chickenpox in the family she developed antibodies against the disease.

(c) 2002 BMJ.





Discuss This Article Post a Comment 




[ Be the first to comment on this article ]




 
Free Chronic Fatigue Syndrome and Fibromyalgia Newsletters
Subscribe to
Our FREE
Newsletter
Subscribe Now!
Receive up-to-date ME/CFS & Fibromyalgia treatment and research news
 Privacy Guaranteed  |  View Archives

Featured Products

Optimized Curcumin Longvida® by ProHealth Optimized Curcumin Longvida® by ProHealth
Supports Cognition, Memory & Overall Health
Energy NADH™ 12.5mg Energy NADH™ 12.5mg
Improve Energy & Cognitive Function
Mitochondria Ignite™ with NT Factor® Mitochondria Ignite™ with NT Factor®
Reduce Fatigue up to 45%
FibroSleep™ by ProHealth FibroSleep™ by ProHealth
The All-in-One Natural Sleep Aid
Ultra EPA  - Fish Oil Ultra EPA - Fish Oil
Ultra concentrated source of essential fish oils

Natural Remedies

The Most Powerful Natural Antioxidant Discovered to Date - Hydroxytyrosol The Most Powerful Natural Antioxidant Discovered to Date - Hydroxytyrosol
Anti-Inflammatory Properties of Tart Cherry Anti-Inflammatory Properties of Tart Cherry
Three-Step Strategy to Reverse Mitochondrial Aging Three-Step Strategy to Reverse Mitochondrial Aging
Are You Obtaining the Proper Enzymes? Are You Obtaining the Proper Enzymes?
Protect Against Sun-Induced Skin Aging From The Inside Out Protect Against Sun-Induced Skin Aging From The Inside Out

FIBROMYALGIA RESOURCES
What is Fibromyalgia?
Fibromyalgia 101
Fibromyalgia Symptoms
Fibromyalgia Treatments
| CFS RESOURCES
What is CFS?
ME/CFS 101
ME/CFS Symptoms
ME/CFS Treatments
| FORUMS
Fibromyalgia
ME/CFS
ADVANCED MEDICAL LABS
WHOLESALE  |  AFFILIATES
GUARANTEE
CONTACT US
PRIVACY
RSS
SITE MAP
ProHealth on Facebook  ProHealth on Twitter  ProHealth on Pinterest  ProHealth on Google Plus
Credit Card Processing