rAAV vector engineering
Construction, large-scale production, and in vivo application of tyrosine-mutant rAAV vectors with cell-specific promoters and engineered capsids to enhance transduction and cell-type specificity in the retina.
What is gene therapy
Genetic instruction delivered as treatment
Gene therapy is the treatment of disease at the molecular level: it transfers specific genetic material to target cells for therapeutic purposes, restoring lost functions or modifying pathogenic mechanisms.
At LTGVV we use vectors derived from the recombinant adeno-associated virus (rAAV) — non-pathogenic, low-immunogenicity vectors with prolonged transgene expression. We combine capsid molecular engineering, CRISPR-Cas9, and mitochondrial transplantation to investigate and treat neurodegenerative diseases, with a focus on glaucoma and retinal dystrophies.
Research lines
Molecular engineering for advanced therapies
Neuroprotective gene therapy for glaucoma
Therapeutic strategies with neuroprotective transgenes (MAX, BIP, p22phox, CHIP, PEDF) to rescue retinal ganglion cells in preclinical models of acute and chronic glaucoma.
CRISPR-Cas9 in gene therapy
Development of advanced therapy products based on CRISPR-Cas9 delivered by rAAV vectors for permanent insertion of neuroprotective transgenes in retinal ganglion cells.
Mitotherapy: mitochondrial transplantation
Investigation of the neuroprotective and pro-regenerative potential of isolated active mitochondria transplantation in models of glaucomatous degeneration and optic nerve injury.
Retinal transcriptomics
Transcriptomic analysis of the retina treated by gene therapy in glaucoma models to identify gene networks involved in neuroprotection and pathogenic mechanisms.
Advanced therapy regulation
Active participation in the National Network of Advanced Therapy Experts (RENETA), supporting ANVISA in the technical evaluation of gene therapy products in Brazil.
Funding
Research funded by Brazilian agencies
