Lentivirus Success May Herald Gene Therapy for Glaucoma

Researchers have demonstrated a method to permanently transplant genes into the trabecular meshwork of the eye in a first step toward the development of a cure for glaucoma.

Glaucoma affects about 70 million people worldwide, many over age 65. It is a chronic disease that now is treated with surgery and then daily eye drops for the rest of the patient's life.

Investigators at the Mayo Clinic (Rochester, MN, USA) used a lentivirus vector to deliver a jellyfish gene coding for a green fluorescent protein into the trabecular meshwork tissue in the eyes of a group of domestic cats. They reported in the September 2004 issue of Investigative Ophthalmology and Visual Science that noninvasive observation revealed that the cats' eye tissues produced green proteins and that this production persisted for more than 10 months.

Senior author Dr. Eric Poeschla, a virologist at the Mayo Clinic, explained, "The results meet three criteria that have represented a main hurdle: enough delivery to enough cells; targeted delivery to the relevant cells; and permanence. The main message here is that a specific kind of gene vector--a lentiviral vector--could be engineered to work really well as a delivery system to the particular tissue involved in glaucoma. The next challenge is to replace the jellyfish protein with one that can safely treat glaucoma, which is what we are trying to accomplish now.”



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