Navigation Links
Jeffrey Modell Foundation supports Belgian research on primary immunodeficiency
Date:9/9/2014

For the first time the Jeffrey Modell Foundation is giving a research grant to a Belgian laboratory. The team of Adrian Liston from VIB-KU Leuven will use the grant to develop a gene therapy to cure children that suffer from IPEX syndrome, a rare and fatal autoimmune disorder in which the immune system attacks the body's own tissues and organs. At the moment, the only successful therapy to treat the syndrome is a bone marrow transplantation, which is not available for all children.

"This is a real chance for a cure", said group leader Adrian Liston. "The gene responsible for this disease was identified 13 years ago, but for the first time we may have learned enough about the basic biology to solve it. We should know within a year whether the gene therapy works in mice, after which we can move to patients at top speed."

The Jeffrey Modell Foundation (JMF)

JMF is a global non-profit organization for patients who suffer from Primary Immunodeficiency (PI) and their relatives. The organization is devoted to early and precise diagnosis, meaningful treatments and, ultimately, cures. Through clinical and basic research, physician education, patient support, advocacy, public awareness and new-born screening they want to make a difference in the lives of patients with PI.

Vicki and Fred Modell established the Foundation in 1987, in memory of their son Jeffrey, who died at the age of fifteen from complications of PI. During the years, the foundation has created a network of the world's leading expert immunologists. Two years ago the Child Immune Deficiencies Department of UZ Leuven was given the first certification as a "Jeffrey Modell Foundation Diagnostic and Research Center for Primary Immunodeficiencies" in Belgium.

IPEX and primary immunodeficiency (PI)

IPEX is an acronym for immune dysregulation, polyendocrinopathy (diseases affecting multiple endocrine glands), enteropathy (disorder of the intestines), and X-linked (pattern of inheritance).

IPEX Syndrome is classified as a primary immunodeficiency disorder. Primary immunodeficiencies are disorders in which part of the body's immune system is missing or does not function normally. IPEX is caused by mutations in the FOXP3 gene which lead to the dysfunction of regulatory T cells (a type of white blood cells).

IPEX syndrome is an autoimmune disorder, meaning that the immune system mistakenly attacks the body's own tissues and organs. The syndrome is characterized by severe diarrhoea, dermatitis (inflammation of the skin), diabetes and severe, life-threatening infections. The disease only affects boys.

Current therapies still remain of partial efficacy. Immunosuppressive drugs are most commonly used, but they only delay the disease. Stem cell transplantation, when performed before severe autoimmunity develops, is currently the only effective cure. However transplantation is only a solution for those children with a compatible donor, unless a gene therapy option is available to correct the mutation in the patient's own stem cells.


'/>"/>

Contact: Evy Vierstraete
info@vib.be
32-924-46611
VIB (the Flanders Institute for Biotechnology)

Source:Eurekalert

Related biology news :

1. TGen President Dr. Jeffrey Trent speaks at Brookings Institution biomedical conference
2. Phoenix Community Alliance honors TGens Dr. Jeffrey Trent
3. Study explores the role of uncertainty in infectious disease modelling
4. International Balzan Foundation announces 2014 prize winners
5. Hartman Foundation expands support for co-op program
6. Biometrics UnPlugged Welcomes the Electronic Frontier Foundations Jennifer Lynch to the Mobility at the Crossroads of Commerce and Privacy Summit
7. Kessler Foundation researchers publish first study of brain activation in MS using fNIRS
8. Berger Foundation funds innovative scholarship at UCR School of Medicine
9. Rossi Foundation pledges $1M for JAX neurobehavioral research center
10. The Brain & Behavior Research Foundation names winners of Klerman and Freedman Prizes
11. Albert Einstein College of Medicine receives grant from the Michael J. Fox Foundation to fund drug discovery project targeting Parkinsons
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/23/2019)... ... May 23, 2019 , ... Federal Publications Seminars, the ... is pleased to announce the release of FPSOnline 2.0, an updated version of its ... weeklong conferences , La Jolla Government Contracts Week, were given a “sneak peek” ...
(Date:5/22/2019)... (PRWEB) , ... May 22, 2019 , ... ... built, helium-based leak testing instruments for the Pharmaceutical, Biotechnology, Medical Device ... agreement with Industrial Production Processes (IPP) Ltd has been signed. ...
(Date:5/21/2019)... ... 2019 , ... Suvoda LLC , a SaaS provider of Interactive Response ... one of Inc. Magazine’s Best Workplaces for 2019, Inc.’s fourth annual ranking of the ... and hitting the newsstands on May 28, is the result of a comprehensive measurement ...
Breaking Biology News(10 mins):
(Date:6/11/2019)... ... June 10, 2019 , ... DeepDyve and ... journals to DeepDyve’s rental service for peer-reviewed journals. , IOPP’s portfolio represents ... than 20 million articles, sourced from more than 15,000 journals. , “By partnering ...
(Date:6/11/2019)... N.C. (PRWEB) , ... June 11, 2019 , ... ... extracellular vesicles derived from human mesenchymal stem cells (MSC-EV) are able to incorporate ... cloning ability. In a test on mice, MSC-EVs also increased the cells’ ability ...
(Date:5/31/2019)... ... May 29, 2019 , ... ... from the USPTO providing proprietary interest to our methodology, processes, and diagnostic ... DIAGNOSIS AND TREATMENT… extends Somnology’s IP rights including our proprietary sleep scoring ...
(Date:5/31/2019)... ... May 30, 2019 , ... A study released ... delivery system for the sustained release of human placental stem cell (HPSC)-derived conditioned ... able to deliver CM into the injured kidney, where it helped restore function ...
Breaking Biology Technology: