Activate Now
 
ProHealth health Vitamin and Natural Supplement Store and Health
Home  |  Log In  |  My Account  |  View Cart  View Your ProHealth Vitamin and Supplement Shopping Cart
800-366-6056  |  Contact Us  |  Help
Facebook Google Plus
Fibromyalgia  Chronic Fatigue Syndrome & M.E.  Lyme Disease  Natural Wellness  Supplement News  Forums  Our Story
Store     Brands   |   A-Z Index   |   Best Sellers   |   New Products   |   Deals & Specials   |   Under $10   |   SmartSavings Club

Trending News

Acupuncture used in clinical settings reduced symptoms of menopause

Benefits of Coconut Oil Are Undeniable

How Awesome Is Ashwagandha?

SURVEY RESULTS: Should Opioids Be Restricted for Chronic Illness?

CoQ10 supplementation associated with lower pro-inflammatory factors in randomized trial

Clinical trial demonstrates success of low FODMAP diet

Research Demonstrates Superiority of Krill Oil Compared to Fish Oil

Vitamin nicotinamide riboside protects mice from diabetes complications

Vitamin-mineral supplement prevents brain cell loss in recent research

What’s so Remarkable About Rosemary?

 
Print Page
Email Article

Scientists Use Gene Therapy to Cure Immune Deficient Child

  [ 11 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.



Post a Comment

Featured Products From the ProHealth Store
Vitamin D3 Extreme™ FibroSleep™ Ultra ATP+, Double Strength

Looking for Vitamins, Herbs and Supplements?
Search the ProHealth Store for Hundreds of Natural Health Products


Article Comments



Be the first to comment on this article!

Post a Comment


 
All Natural Deodorants

Featured Products

Energy NADH™ 12.5mg Energy NADH™ 12.5mg
Improve Energy & Cognitive Function
Optimized Curcumin Longvida® Optimized Curcumin Longvida®
Supports Cognition, Memory & Overall Health
Mitochondria Ignite™ with NT Factor® Mitochondria Ignite™ with NT Factor®
Reduce Fatigue up to 45%
Ultra EPA  - Fish Oil Ultra EPA - Fish Oil
Ultra concentrated source of essential fish oils
Ultra ATP+, Double Strength Ultra ATP+, Double Strength
Get energized with malic acid & magnesium

Natural Remedies

Green Coffee Extract: Unique Obesity Intervention Green Coffee Extract: Unique Obesity Intervention
Magnesium: An Essential Supplement for ME/CFS Magnesium: An Essential Supplement for ME/CFS
How Glutathione Can Save Your Life How Glutathione Can Save Your Life
Aches and Pains? A Simple Solution You'll Love Aches and Pains? A Simple Solution You'll Love
Block food Cravings At Their Molecular Root Block food Cravings At Their Molecular Root

CONTACT US
ProHealth, Inc.
555 Maple Ave
Carpinteria, CA 93013
(800) 366-6056  |  Email

· Become a Wholesaler

· Affiliate Program
SHOP WITH CONFIDENCE
Credit Card Processing
SUBSCRIBE AND SAVE 15% NOW*
Be the first to know about new products, special discounts and the latest health news. *New subscribers only

CONNECT WITH US ProHealth on Facebook  ProHealth on Twitter  ProHealth on Pinterest  ProHealth on Google Plus

© 2016 ProHealth, Inc. All rights reserved. Store  |  Customer Service  |  Guarantee  |  Privacy  |  Contact Us  |  Library  |  RSS  |  Site Map