Wiskott Aldrich Syndrome News and Research

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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.

Study reveals the mechanism behind oligomeric assembly of a BAR-domain-containing protein

Cell membranes play a critical role by serving as containment units and separating the inner cellular space from the extracellular environment.

26 Apr 2023

New underlying cause of Wiskott-Aldrich syndrome discovered

Researchers at the Salk Institute and King Abdullah University of Science and Technology (KAUST) in Saudi Arabia have discovered a new underlying cause of Wiskott-Aldrich syndrome, a rare genetic disease that leads to bleeding and immune deficiencies in babies.

13 Jul 2022

Study yields molecular clues that could lead to new treatments for rare genetic disease

A new stem cell study by KAUST researchers helps to explain a rare genetic disease called Wiskott-Aldrich syndrome (WAS), yielding molecular clues that could lead to new treatments for a devastating immune deficiency disorder.

6 Jul 2022

Newly identified pathway controls dangerous overreactions in the body's immune system

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.

26 Mar 2021

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

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.

2 Jun 2020

Clinical cell therapy expert shares the benefits of donating cord blood

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.

29 Jul 2019

Using novel gene therapy to cure infants born with severe combined immunodeficiency

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.

29 Aug 2018

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

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.

4 Oct 2017

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

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.

12 Oct 2016

Gene therapy shows promise in children with Wiskott-Aldrich syndrome

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).

7 Dec 2015

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

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.

7 Dec 2015

International researchers identify previously unknown genetic immunodeficiency

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.

19 Jun 2015

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

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

29 Apr 2015

Gene therapy reduces infectious complications in children, teens with Wiskott-Aldrich syndrome

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.

22 Apr 2015

Researchers develop adjuvant which enhances effect of virus transduction

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.

5 Mar 2014

Imaginative play with medically themed toys may improve recovery in chronically ill children

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.

6 Nov 2013

Stem cell lines are ideal research tools for designing models to understand disease progression

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.

25 Sep 2013