Tag: gene therapy

A new narrative review outlines how hyaluronidase can transiently open the hyaluronic acid-rich extracellular matrix to enhance nose-to-brain delivery of ...

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 ...

A University of Cincinnati and UC Davis team showed that an AAV gene therapy targeting cerebrospinal fluid-producing cells reduced hydrocephalus-related ...

MIT researchers have engineered ligand-directed lipid nanoparticles that reprogram natural killer cells into CAR-bearing cancer killers directly inside living animals, ...

A new Pediatric Research reflection by Alex Smith traces nearly twenty years of living alongside his son Harrison's Duchenne muscular ...