Severe Combined Immunodeficiency News and Research

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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

Risk factors for severe RSV infection in immunocompromised children

St. Jude Children's Research Hospital investigators and collaborators have shown how to predict if a child who is infected with respiratory syncytial virus (RSV) while being treated for cancer or another catastrophic disease is at high risk for developing severe infection. The finding will help clinicians improve guidelines for managing these infected children.

4 Feb 2008

Debate over safety of gene therapy trials focuses on issue of informed consent

Can a patient who agrees to participate in a safety study of a gene therapy protocol give truly informed consent and understand the risks involved when the consent forms are highly technical and the physician or institution seeking their consent has a stake in the study and its outcome?

23 Jan 2008

Boy in a bubble gets leukaemia after gene therapy

A child who was born with a rare immunodeficient condition has developed leukaemia as a result of gene therapy he received at Great Ormond Street children's hospital in London.

19 Dec 2007

Novel technologies show promise for future of gene transfer

Several new technologies that could significantly impact the future of gene transfer applications were presented at a conference at the 10th Annual Meeting of the American Society of Gene Therapy (ASGT) in Seattle.

3 Jun 2007

Researchers determine why gene therapy treatment caused leukemia in some X-linked severe combined immunodeficiency patients

Scientists at St. Jude Children's Research Hospital have developed a mouse model of a severe disease of the immune system that helps explain why gene therapy used to treat children with this disease at an institution in Europe caused some of them to develop leukemia.

2 Aug 2006

Researchers develop T-cells from human embryonic stem cells

Human embryonic stem cells can be genetically manipulated and coaxed to develop into mature T-cells, raising hopes for a gene therapy to combat AIDS.

3 Jul 2006

Therapeutic gene suspected to cause cancer

One of the few gene therapy studies that had ever been successful was terminated in 2003, because several of the treated children had developed leukemia.

3 May 2006

Nod1 found to control tumor growth in certain breast cancers

This protein was previously thought to play a role solely in the innate immune system's response to bacterial infection. In the new study, which will be published on-line by the Proceedings of the National Academy of Sciences

12 Feb 2006

Dental problems caused by radiation, chemotherapy should be treated before a child undergoes immunosuppression as part of bone marrow transplantation therapy

Many children who undergo bone marrow transplantation (BMT) as part of cancer treatment already have dental abnormalities that leave them vulnerable to potentially life-threatening bacterial infections, according to investigators at St. Jude Children's Research Hospital. A report on this study appears in the prepublication online edition of Bone Marrow Transplantation.

29 Aug 2005