Advanced Cell and Gene Therapy

Gene Editing Technologies: CRISPR and Beyond

Gene therapy is emerging as a transformative approach for treating a variety of ophthalmic diseases, offering the potential to correct genetic defects, halt disease progression, and restore vision. Ophthalmic diseases, such as inherited retinal dystrophies, age-related macular degeneration (AMD), and glaucoma, can significantly impact quality of life, and gene therapy provides a promising avenue for long-term treatment and possible cures. Employing RNA interference (RNAi) or antisense oligonucleotides to silence the expression of harmful genes. This is useful for conditions caused by gain-of-function mutations or toxic protein production. Gene therapy research for RP involves various approaches, including gene augmentation, gene editing, and optogenetics, targeting different genetic mutations associated with the disease. Gene therapy for glaucoma focuses on neuroprotection and lowering intraocular pressure. This includes delivering genes that protect retinal ganglion cells from degeneration or enhance the outflow of aqueous humor to reduce pressure.