Tag: Gene Therapy

A cross-sectional survey of 53 Spanish healthcare professionals found high willingness to adopt Duchenne muscular dystrophy gene therapy, alongside concerns ...

A mouse study links high-fat feeding to reduced PQBP1, altered synaptic-gene RNA splicing, and impaired cognition while showing that restoring ...

A CD276-targeted antibody-drug conjugate combined with mitochondrial-disrupting gene therapy outperformed either treatment alone in two preclinical models of metastatic triple-negative ...

A study found anti-AAV3B neutralizing antibodies in 33%–36% of healthy adults, with intermediate titers in 19% of positive individuals, while ...

UCLA scholars Susan Ettner, Donald Kohn and Dennis Slamon were elected to the National Academy of Medicine for work on ...

Restoring cardiac METTL14 reduced inflammation and fibrosis while improving survival in two mouse models of progeria.

UCLA physician-scientist Donald Kohn’s election to the National Academy of Medicine honors decades of work advancing blood stem cell gene ...

Blood vessels do more than carry blood: the endothelial cells lining them regulate inflammation, blood flow, tissue repair and the ...

New research in cystic fibrosis rats shows that chronic Pseudomonas aeruginosa lung infection does not hinder lentiviral vector gene transfer ...

Researchers report a non-viral, OCT-guided method using gold nanorods and a 980-nm laser to deliver the opsin MCO-020 to targeted ...

A new Oncoscience perspective maps the 2025–2026 glioblastoma clinical trial pipeline across four mechanistic domains, showing how biomarker-guided selection and ...

Infant neuroblastoma is one of the most clinically paradoxical cancers in medicine. Diagnosed during the first year of life, it ...

A sweeping narrative review charts how molecular genetics turned non-malignant blood disorders into the proving ground for modern diagnostics and ...

A McGill University review finds that the ethics of pediatric first-in-human cell and gene therapy trials rests on two foundational ...

Researchers established a stable human packaging cell line producing high-titer γ-retroviral vectors pseudotyped with the non-fusogenic BaEV-TR envelope protein, overcoming ...

Researchers used a vector-free CRISPR activation system to boost dopamine biosynthesis genes in cellular models of Parkinsonian neurotoxicity, finding modest ...

A comprehensive review details how three FDA-approved therapies have dramatically improved spinal muscular atrophy outcomes while highlighting persistent comorbidities and ...

A mouse study shows that the scaffolding protein rapsyn is depleted after tourniquet-induced ischemia-reperfusion injury, shattering acetylcholine receptor clusters at ...

Researchers have built a single-nucleus transcriptomic atlas of the developing human inner ear, revealing that Hedgehog signaling repression is required ...

Harvard-led researchers developed a transcriptomic aging clock to screen rejuvenation genes and found that over-expressing the RNA splicing factor SRSF1 ...