Severe Combined Immunodeficiency (SCID) News and Research

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Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening performed in numerous states indicates that the incidence of the potentially life-threatening disorder, severe combined immunodeficiency, is higher than previously believed, at 1 in 58,000 births, although there is a high rate of survival, according to a study in the August 20 issue of JAMA.

20 Aug 2014

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Babies who are born with severe combined immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, according to experts led by Memorial Sloan Kettering's Richard J. O'Reilly, MD, a world-renowned pioneer in the development of transplant protocols.

31 Jul 2014

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

Two pediatricians whose research led to the need for early identification of and treatment of severe combined immunodeficiency (SCID) and its inclusion in newborn screening have received the March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics.

29 Mar 2014

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

A series of advancements in genetically engineered cell therapies demonstrate early efficacy and safety in patients with blood disorders for whom standard treatments have been unsuccessful, according to data showcased today during the 55th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans.

9 Dec 2013

UCSF to receive grant to study gene sequencing aimed at detecting disorders in newborns

UC San Francisco will receive $4.5 million over the next five years for a pilot project to assess whether large-scale gene sequencing aimed at detecting disorders and conditions can and should become a routine part of newborn testing.

6 Sep 2013

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

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

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

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

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

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

Severe Combined Immunodeficiency (SCID) News and Research

Newborn screening indicates that incidence of SCID is higher than previously believed

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

UCSF to receive grant to study gene sequencing aimed at detecting disorders in newborns

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

Refinements in gene therapy prove beneficial for ‘bubble babies’

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

FTY720 improves locomotor recovery in mice with SCI

Severe combined immunodeficiency treated successfully with gene therapy

Researchers find temperature-sensing protein in immune cells

Study suggests newborn diagnosis can improve survival in SCID patients

SCID added to the core panel of 29 genetic disorders

Statewide blood screening program identifies infants with T-cell lymphopenia

Discovery of new genetic cause severe combined immunodeficiency (SCID)

Boy in a bubble gets leukaemia after gene therapy

Nod1 found to control tumor growth in certain breast cancers

Gene therapy effective in helping children with severe combined immunodeficiency (SCID)

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