420 articles in this topic
Researchers engineer tissue-targeting lipid nanoparticles that precisely deliver mRNA and prime editing tools to lungs, liver, spleen, thymus, and bone.
Computational protein design produces potent GPCR-targeting miniproteins for pain, cancer, obesity, and migraine with in vivo efficacy.
Wave Life Sciences shares early trial data on an RNA editing treatment for alpha-1 antitrypsin deficiency, targeting monthly dosing.
A second wave of RAS-targeting therapies is reshaping cancer treatment, with implications for longevity through better tumor control.
A comprehensive review of pathophysiology, genetics, and emerging therapies for mitochondrial myopathies, spotlighting elamipretide and gene therapy.
A phase 2b trial finds allogeneic MSC infusions improved 6-minute walk distance by 63 meters vs placebo at 9 months in frail older adults.
AstraZeneca's baxdrostat wins FDA approval for hypertension, offering a novel mechanism for patients uncontrolled on existing therapies.
A Phase 2 trial evaluates MBA-P01, a novel botulinum toxin A, for safety and optimal dosing in treating frown lines.
Cytosine base editing achieved 62–89% efficiency fixing a hyperinflammatory disease gene, but revealed distinct genotoxicity risks across cell types.
BIIB080 failed its primary endpoint in Phase 2, yet Biogen sees potential signals worth pursuing in the fight against Alzheimer's tau tangles.
Harvard-developed ENTER nanoparticles outperform lipid transfection reagents in delivering mRNA, CRISPR, proteins, and siRNA to diverse cell types.
A special issue overview surveys three decades of peptide-based materials research, spotlighting applications from drug delivery to regenerative medicine.
