Severe Combined Immunodeficiency News and Research

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Orphan, St George's sign licensing agreement to develop new therapies for metabolic disorders

St George's, University of London has signed an exclusive worldwide licensing agreement with rare-disease research-and-development firm Orphan Technologies Ltd to develop new therapies for deadly metabolic disorders.

5 Feb 2013

Refinements in gene therapy prove beneficial for ‘bubble babies’

Results from a long-term study published in Blood show that adding a low dose of chemotherapy prior to gene therapy can help improve outcomes in children with adenosine deaminase-deficient severe combined immunodeficiency.

17 Sep 2012

Gene therapy regimen can safely restore immune systems to children with Bubble Boy disease

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

12 Sep 2012

Gene therapy can effectively treat ADA-deficient severe combined immunodeficiency

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response.

25 May 2012

Collaborative research discovers genetic marker that identifies pigs with reduced susceptibility to PRRS

A collaborative discovery involving Kansas State University researchers may improve animal health and save the U.S. pork industry millions of dollars each year.

16 Mar 2012

FTY720 improves locomotor recovery in mice with SCI

A new study suggests that administering FTY720, an oral drug that has shown promise in trials for human multiple sclerosis, significantly improves locomotor recovery in mice with spinal cord injury.

13 Mar 2012

New inexpensive medical sensor may simplify diagnosis of diseases

An inexpensive new medical sensor has the potential to simplify the diagnosis of diseases ranging from life-threatening immune deficiencies to the common cold, according to its inventors at the Stanford University School of Medicine.

8 Mar 2012

Severe combined immunodeficiency treated successfully with gene therapy

Gene therapy can offer a cure for a rare condition that causes children’s immune systems to fail. In this rare condition, known as Severe Combined Immunodeficiency (SCID), inherited genetic mutations mean that babies are unable to fight off infection, severely limiting their chances of surviving more than a few years. Severe combined immunodeficiency (SCID) is a rare genetic disorder estimated to affect between 1 in 200,000 and 1 in 1,000,000 live births

26 Aug 2011

Global gene therapy market to reach $300 million by 2015

The global gene therapy market is expected to grow to over $300 million by 2015, according to a new report available on companiesandmarkets.com. The gene therapy market will grow further driven by the human genome project, the increasing incidence of cancer and other critical diseases.

6 Jun 2011

Researchers find temperature-sensing protein in immune cells

Researchers at The Scripps Research Institute have found a temperature-sensing protein within immune cells that, when tripped, allows calcium to pour in and activate an immune response.

7 May 2011

Study suggests newborn diagnosis can improve survival in SCID patients

Severe Combined Immunodeficiency occurs in just one out of every 50,000 to 100,000 births in the United States, yet it is the most serious primary immunodeficiency disorder. A study published today in Blood, the Journal of the American Society of Hematology, demonstrates that babies with SCID who are diagnosed at birth and receive a hematopoietic stem cell transplant, which is the transplantation of blood-forming stem cells, have significantly improved survival.

28 Jan 2011

Cincinnati Children's Hospital Medical Center named Centers of Excellence for Molecular Hematology

Cincinnati Children's Hospital Medical Center has been named one of five national Centers of Excellence for Molecular Hematology to find new gene and cell therapies for inherited diseases affecting blood cells.

8 Oct 2010

SCID added to the core panel of 29 genetic disorders

On May 21, 2010, Kathleen Sebelius, Secretary of Health and Human Services announced the addition of Severe Combined Immunodeficiency -- commonly known as bubble boy disease -- to the core panel of 29 genetic disorders -- as part of her recommendation to adopt the national Recommended Uniform Screening Panel.

26 May 2010

Special issue reviews current state of science in fetal therapy

Repairing birth defects in the womb. Inserting a tiny laser into the mother's uterus to seal off an abnormal blood flow and save fetal twins. Advancing the science that may allow doctors to deliver cells or DNA to treat sickle cell anemia and other genetic diseases before birth.

17 Feb 2010

Statewide blood screening program identifies infants with T-cell lymphopenia

The testing of DNA from a statewide blood screening program for newborns in Wisconsin was able to identify infants with T-cell lymphopenia, a blood disorder that affects the child's immune system, a disease in which early identification is important, according to a study in the December 9 issue of JAMA.

9 Dec 2009

FDA-approved drug PEG-ADA shows positive results in relieving major complication of priapism

Thousands of men are afflicted with an embarrassing and painful condition that triggers spontaneous, long-lasting erections. There are limited treatment options, but a solution could be on the way thanks to new research at The University of Texas Health Science Center at Houston.

31 Oct 2009

Roche's high-throughput sequencing technology used for high-resolution HLA genotyping

A study published this month in the journal Tissue Antigens reports that researchers at Roche Molecular Systems in Pleasanton, CA have used high-throughput sequencing technology from 454 Life Sciences, a Roche Company, to carry out high-resolution human leukocyte antigen (HLA) genotyping in research samples.

27 Oct 2009

NIH funds the University of California's consortium for immune disorder research

The University of California, San Francisco has been designated to lead a new consortium that will study a group of severe immune disorders known as primary immunodeficiencies and aims to improve treatment for these often life-threatening diseases. The Primary Immune Deficiency Treatment Consortium comprises 13 centers throughout the United States and has a $6.25 million funding commitment over five years from the National Institutes of Health.

13 Oct 2009

Discovery of new genetic cause severe combined immunodeficiency (SCID)

Severe combined immunodeficiency (SCID) is an inherited disease sometimes known as 'Boy in the bubble syndrome', because the patient lacks one or more type of immune cell, making them very susceptible to infections.

15 Dec 2008

Findings may aid in bone marrow transplants, blood diseases

New research sheds light on the biological events by which stem cells in the bone marrow develop into the broad variety of cells that circulate in the blood. The findings may help improve the success of bone marrow transplants and develop better treatments for life-threatening blood diseases.

5 Aug 2008