Wiskott-Aldrich Syndrome is an inherited immune disorder that occurs in young boys. It causes eczema (a type of skin inflammation), a decrease in the number of platelets (blood cells that help prevent bleeding), and frequent bacterial infections. People with Wiskott-Aldrich syndrome are at increased risk of developing leukemia and lymphoma. Also called Aldrich syndrome.
Scientists at Sinai Health say they have discovered a new pathway that controls dangerous overreactions in a body's immune system, including deadly forms of hyper-inflammation.
Seattle Children's Research Institute, one of the top pediatric research institutions in the world, and global biotechnology leader CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases.
July is National Cord Blood Awareness Month, and many parents are not aware of the option they have to donate it and the benefits that public donation can bring.
Out of every 60,000 births, a baby arrives to face the world without a fully functioning immune system leaving them unequipped to fight even the most common infections.
In a recent clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy (CALD) -- a neurodegenerative disease that typically claims young boys' lives within 10 years of diagnosis -- effectively stabilized the disease's progression in 88 percent of patients, researchers from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Massachusetts General Hospital report today.
A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients.
Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology (abstract #260).
New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer.
An analysis of five families has revealed a previously unknown genetic immunodeficiency, says an international team led by researchers from Boston Children's Hospital. The condition, linked to mutations in a gene called DOCK2, deactivates many features of the immune system and leaves affected children open to a unique pattern of aggressive, potentially fatal infections early in life.
Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy
In a small study that included seven children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder, use of gene therapy resulted in clinical improvement in infectious complications, severe eczema, and symptoms of autoimmunity, according to a study in the April 21 issue of JAMA, a theme issue on child health.
Lentiviruses, which belong to the family of retroviruses, are used as vectors to exchange genetic material in cells and can be used to replace a defective gene as defined by gene therapy.
New research finds that chronically ill children celebrate a successful recovery. It's through their imaginative play with medically themed toys. Laura Nabors, an associate professor of human services in the University of Cincinnati College of Education, Criminal Justice, and Human Services (CECH), will present new research on Tuesday, Nov. 5, at the American Public Health Association's (APHA) 141st Annual Meeting and Expo in Boston.
Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA.
Researchers are having success using a "defanged" version of HIV to deliver gene therapy to children suffering from genetic diseases, a development that could improve other such care.
Today's early morning highlights from the major news organizations, including articles on data tracking of doctors and comparing the rollout of the Medicare Part D drug plans to implementation of the federal health law.
Mycobacterium ulcerans infects the skin and subcutaneous tissues and secretes a lipid toxin, mycolactone, which causes open skin lesions, known as Buruli ulcers.
Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] as a treatment for multifocal motor neuropathy (MMN). This is the first immunoglobulin treatment approved for MMN patients in the United States, and it was approved for use with MMN patients in Europe in 2011.
Baxter International Inc. today provided an update on its clinical program evaluating the use of its GAMMAGARD LIQUID 10%, for the treatment of mild to moderate Alzheimer's disease.
Baxter International Inc. today announced that the company has submitted a supplemental biologics license application (sBLA) to the U.S. Food and Drug Administration (FDA) for approval of GAMMAGARD LIQUID 10% for the treatment of multifocal motor neuropathy.