Health ❯ Therapeutics

Gene Therapy

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

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

Peer‑reviewed results from Mass General Brigham precede an IND submission under FDA guidance.

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