Health ❯ Therapeutics

Gene Therapy

Gene Editing Lentiviral Therapy RNA-Based Therapies Mitochondrial Gene Therapy CSF1R Mutations Stem Cell Therapy Neuroprotection Cell Replacement Therapy Cocaine Treatment RNA-based Therapies Viral Vectors VMAT2 Cellular Therapy Genome Editing Protein Regulation Cellular Regeneration

Study Shows Adult Mouse Brains Replace Lost Astrocytes by Moving Cell Nuclei

Mapping of the repair process identifies transient gene programs that suggest drug targets while leaving human relevance untested.

CRISPR Enzyme Cas12a2 Selectively Destroys Cells Based on RNA in Lab Tests

Custom CRISPR Base Editor Corrects ACTA2 Mutation in Mice, Extending Survival Fourfold

ALS Study Identifies 39-Gene Vulnerability Signature and Cross-Species Biomarkers

Brain-Specific Microglia Replacement Shows Durable Benefits in Sandhoff Mouse Models

Zebrafish Cyclin D Genes Reveal Pathway for Mammalian Hearing Restoration

Microglia Replacement Therapy Halts Progression in ALSP Patients