Prime-editing therapy targets PRPH2 splice mutations (c.828+) for retinal dystrophies, enabling precise in-eye gene corrections.

AAV-based gene therapy reactivates Islet1 and Lhx3 to boost motor neuron resilience and potentially slow ALS progression.

Mutation-independent CRISPR pair replaces dominant alleles with a functional gene to treat autosomal dominant diseases.

Selective peptide nucleic acid polymers block cancer gene expression, targeting mutations like BRAFV600E with improved delivery and specificity.

Precise Hspa8 variant enhances synaptic SNARE function to treat SMA and other neurodegenerative diseases, via gene therapy.

AAV BEST1 gene therapy targets dominant BEST1 retinal mutations to restore function and potentially cure associated degeneration.

PPRDecoder: a genome-wide, unbiased algorithm predicting RNA targets of PPR proteins, accelerating gene regulation research.

CRB1-targeted gene therapy delivers isoform-specific CRB1 to Müller glia and photoreceptors, treating CRB1-retinopathies.

Co-delivery platform stabilizes nucleic acid therapeutics with exosome carriers and RISC associations, boosting efficacy while reducing off-targets.

Broad, gene-agnostic retinal therapy using AAV-delivered PGC1α/NRF2 or inhibitors to rebalance metabolism, reduce oxidative stress, slow degeneration.

Spinal motor neuron–specific enhancer enables AAV-deliverable, cell-type–restricted gene overexpression/knockdown in adults and development.

Metabolic reprogramming to boost cellular anabolism, slowing neurodegeneration by targeted glycolytic pathway manipulation.

Anti-CRISPR-based timing control confines Cas9 activity to early embryo stages, reducing mosaicism and off-target edits.

Engineered AAV9 gene therapy replaces TK2 to treat TK2 deficiency mitochondrial diseases, potentially correcting DNA depletion and symptoms.

Prime editing corrects CRB1 mutations in retinal dystrophy, offering isoform-independent gene therapy and research tool potential.