Spanish survey finds high DMD gene-therapy willingness amid readiness gaps
A cross-sectional survey of 53 Spanish healthcare professionals found high willingness to adopt Duchenne muscular dystrophy gene therapy, alongside concerns ...
Tag: gene therapy
High-fat diet impairs mouse cognition through altered RNA splicing
A mouse study links high-fat feeding to reduced PQBP1, altered synaptic-gene RNA splicing, and impaired cognition while showing that restoring ...
Smart bomb combination curbs metastatic triple-negative breast cancer in models
A CD276-targeted antibody-drug conjugate combined with mitochondrial-disrupting gene therapy outperformed either treatment alone in two preclinical models of metastatic triple-negative ...
AAV3B antibodies affect eligibility for liver-targeted gene therapy
A study found anti-AAV3B neutralizing antibodies in 33%–36% of healthy adults, with intermediate titers in 19% of positive individuals, while ...
Three UCLA Faculty Members Join National Academy of Medicine
UCLA scholars Susan Ettner, Donald Kohn and Dennis Slamon were elected to the National Academy of Medicine for work on ...
RNA Methylation Restores Heart Function in Progeria Mice
Restoring cardiac METTL14 reduced inflammation and fibrosis while improving survival in two mouse models of progeria.
Donald Kohn’s Gene Therapy Breakthroughs Earn Medical Academy Honor
UCLA physician-scientist Donald Kohn’s election to the National Academy of Medicine honors decades of work advancing blood stem cell gene ...
MTR4 study links a protein to vascular aging in cells and animals
Blood vessels do more than carry blood: the endothelial cells lining them regulate inflammation, blood flow, tissue repair and the ...
Chronic Lung Infection Surprisingly Sustains Gene Therapy Expression in Cystic Fibrosis Rat Lungs
New research in cystic fibrosis rats shows that chronic Pseudomonas aeruginosa lung infection does not hinder lentiviral vector gene transfer ...
OCT-guided laser and gold nanorods deliver opsin genes to degenerating retinas without viruses
Researchers report a non-viral, OCT-guided method using gold nanorods and a 980-nm laser to deliver the opsin MCO-020 to targeted ...
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Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
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University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
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Bee body mass, pathogens and local climate influence heat tolerance
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Researchers record first-ever images and data of a shark experiencing a boat strike
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Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
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