Liquid fat treatment offers hope for rare childhood disease
A liquid fat medicine has shown significant promise in reversing major health complications of rare degenerative disease Ataxia-telangiectasia (A-T).
Aug 6, 2025
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Immune System Diseases: News and Research on Primary Immunodeficiency Diseases
MeSH tree: C20.673.795
X-SCID gene therapy gives scientists a rare glimpse into early immune system
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life.
Jul 31, 2025
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Immune cells turn into 'mini-Hulks' to push away tissue and make space when migrating
Immune responses rely on the efficient movement of immune cells within the complex and geometrically unpredictable three-dimensional tissues that make up our bodies.
Jul 21, 2025
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FDA approves once-monthly Andembry for hereditary angioedema
The U.S. Food and Drug Administration has approved Andembry (garadacimab-gxii) as the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE).
Jun 21, 2025
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Stress genes clear dead cells, offering new disease insights
A new study from The University of Texas at Arlington details a novel strategy for how the body clears out dead cells during stress, revealing unexpected roles for well-known stress-response genes—a discovery that could help ...
Jun 9, 2025
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Researchers explore role of mechanical force in immune responses of a rare genetic disorder
A firm handshake between proteins on immune cells is important for the body's ability to fight infection. Meanwhile, a weak grip helps explain the poor immune deficiencies caused by a rare genetic disorder.
Dec 10, 2024
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Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
Gene technology detects more conditions than standard newborn screening, study finds
Early results from a study of newborn screening methods show that DNA analysis picks up many more preventable or treatable serious health conditions than standard newborn screening and is favored by most parents who are offered ...
Oct 24, 2024
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CRISPR-Cas9 gene editing trial results support further development as treatment for hereditary angioedema
A single treatment with a CRISPR-Cas9 based gene editing therapy is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful and sudden onset of swelling, ...
Oct 24, 2024
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Study: DOCK8 mutation disrupts helper T cell differentiation via glycolytic pathway regulation
A study, published in the journal MedComm and led by Dr. Panpan Jiang and collaborators at Tongji Medical College, explores the critical role of the dedicator of cytokinesis 8 (DOCK8) gene in the differentiation of helper ...
Oct 9, 2024
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