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:9/17/2019)... ... September 17, 2019 , ... VISTAAR released ADVISORY ... strategy or as re-assurance / 2nd opinion to their internal strategies they formed for ... users when going into new markets, or planning to change their product elements and ...
(Date:9/17/2019)... ... 17, 2019 , ... Last week, Blackrock Microsystems, LLC, Neuro ... Medical University of Warsaw, met at the Presidential Palace of the Republic of ... system as part of an initiative called "Startups in the Palace – Cooperation," ...
(Date:9/17/2019)... ... September 17, 2019 , ... ... the 27th Series of the Australia Society of Implant Dentistry (ASID) Accreditation Courses. ... was part of the Surgical Aspects of Modern Implant Dentistry course. Dr. Kim’s ...
Breaking Biology News(10 mins):
(Date:9/17/2019)... ... September 17, 2019 , ... ... Delaney, Director, Project and Clinical Supplies Management, Asia Pacific (APAC) will present at ... Hotel, Singapore, on Sept. 25-26, 2019. , On Thursday, Sept. 26, at 10:10 ...
(Date:9/17/2019)... ... September 17, 2019 , ... Tucker, a Labrador retriever, was just a ... Tucker was limping and lame on his right hip and elbow. At one year ... called “the worst case the vet had seen.” He was prescribed pain medications, both ...
(Date:9/17/2019)... ... September 17, 2019 , ... ... in chronic diseases, announces today that the U.S. Patent and Trademark Office (USPTO) ... algorithm to rank drug class recommendations for hypertension treatment. , The patent ...
(Date:9/17/2019)... ... September 17, 2019 , ... Jennifer Hermansky and Fang Xie, ... named to Law360’s 2019 Rising Stars. Hermansky and Xie, named an Immigration Rising Star ... submissions, spanning 39 practice areas. The list highlights attorneys “whose legal accomplishments transcend their ...
Breaking Biology Technology: