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An international team of researchers at Great Ormond Street Hospital (GOSH), and University of California, Los Angeles (UCLA) have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency that leaves them without an immune system.
Severe combined immunodeficiency due to adenosine deaminase deficiency, also known as ADA-SCID, is a rare, life-threatening disease that prevents children from living a normal life. It is caused by mutations in the gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system.
Children with ADA-SCID have no immune system and, if left untreated, the condition can be fatal within the first two years of life. Day-to-day activities like going to school or playing with friends can lead to a dangerous infection. Recently, new-born screening for SCID has been implemented in some countries to help diagnose the condition early in life.
First of Its Kind Cystic Fibrosis Gene Expression Atlas Reveals Altered Airway Cells
May 10, 2021
Healthy airways (left) show well-defined layers of ciliated cells (green) and basal stem cells (red). In airways affected by cystic fibrosis (right), the layers are disrupted, and scientists identified a transitioning cell type that combines properties of both stem cells and ciliated cells (red and green in the same cell) [Source: UCLA Broad Stem Cell Research Center/Nature Medicine].
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A multi-institute consortium study reports single-cell transcriptomics data that compares cells lining the airways of cystic fibrosis (CF) patients undergoing transplantation for terminal lung disease with that of previously healthy lung donors in a first-of-its-kind molecular atlas.
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Nature Medicine
Healthy airways (left) show well-defined layers of ciliated cells (green) and basal stem cells (red). In airways affected by cystic fibrosis (right), the layers are disrupted, and scientists identified a transitioning cell type that combines properties of both stem cells and ciliated cells (red and green in the same cell).
A team of researchers from UCLA, Cedars-Sinai and the Cystic Fibrosis Foundation has developed a first-of-its-kind molecular catalog of cells in healthy lungs and the lungs of people with cystic fibrosis.
The catalog, described today in the journal Nature Medicine, reveals new subtypes of cells and illustrates how the disease changes the cellular makeup of the airways. The findings could help scientists in their search for specific cell types that represent prime targets for genetic and cell therapies for cystic fibrosis.
Researchers from the UCLA School of Dentistry have discovered a key molecule that allows cancer stem cells to bypass the body s natural immune defenses, spurring the growth and spread of head and neck squamous cell cancers. Their study, conducted in mice, also demonstrates that inhibiting this molecule derails cancer progression and helps eliminate these stem cells.