MeSH tree: E02.095.301
JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that began with the revolutionary idea that DNA can move itself. Now, his team's latest breakthrough is ...
Dec 17, 2025
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There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved proof-of-principle success with "silence ...
Dec 15, 2025
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As a young medical student in 1975, I walked into a basement lab at the National Institutes of Health in Bethesda, Maryland, to interview for a summer job.
Dec 15, 2025
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For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five years, according to a ...
Dec 10, 2025
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A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing ...
Dec 6, 2025
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Researchers at the Eye Genetics Research Unit at Children's Medical Research Institute (CMRI) are the first in the world to use stem cells to study one of the genetic causes of Leber Congenital Amaurosis (LCA)—a rare condition ...
Nov 20, 2025
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Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available. Among lifestyle factors, physical activity stands out as possibly one of the strongest defenders ...
Nov 18, 2025
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The United States on Friday said it would add its most serious warning label to a gene therapy treatment for a debilitating muscle-wasting disease, and also limit its approved use.
Nov 15, 2025
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Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder.
Nov 14, 2025
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Rice University bioengineers have demonstrated a nonsurgical way to quiet a seizure-relevant brain circuit in an animal model. The team used low-intensity focused ultrasound to briefly open the blood-brain barrier (BBB) in ...
Nov 11, 2025
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