MeSH tree: C18.452.648.398.641
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment of metachromatic leukodystrophy (MLD), ...
Mar 12, 2025
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In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase deficiency (MSD), an ultra-rare ...
Jan 29, 2025
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Researchers at Washington University School of Medicine in St. Louis have described the neurodegeneration that occurs in the nervous system of the bowel in Batten disease, a rare and fatal genetic condition. In their latest ...
Jan 15, 2025
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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.
Gregory and Deborah Macres from California lost their son, Gregory, in 1997, when he was just 4 years old. Little Gregory had a severe form of a rare genetic disorder called Gaucher disease and died after unsuccessful experimental ...
Oct 14, 2024
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People from isolated parts of the U.K. could have variations in their genetic code that increase their chance of developing certain diseases, a study suggests.
Oct 4, 2024
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Scientists have conducted the largest and most diverse study to date on how recessive genetic changes contribute to developmental disorders. They found that most undiagnosed cases that are due to recessive causes are linked ...
Sep 23, 2024
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Researchers from Children's Hospital of Philadelphia (CHOP) have tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, ...
Sep 9, 2024
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Cancer cells seldom start off stealthy. Quite to the contrary, they announce their presence to the immune system by planting chemical red flags right on their membranes. Once alerted, the body's defenses can swoop in, destroying ...
Sep 9, 2024
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About 37 million people in the United States have chronic kidney disease and studies show that genetics may explain between 10% and 20% of cases in adults (and as many as 70% of cases in children).
Jul 29, 2024
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