Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly. Positive results in animal models of human retinal disease continue to emerge, as reported in several articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available free on the Human Gene Therapy website at http://www.liebertpub.com/hum.
After 20 years of promising research, testing in animal models, and initial clinical trial results of gene therapy to treat retinal dystrophies, Robin Ali, University College London Institute of Ophthalmology (UK) is "optimistic about the future prospects for retinal gene therapy" mainly because the retina has proven to be a good and accessible target for gene delivery, and scientists have developed efficient and safe viral delivery systems that can successfully introduce therapeutic genes into photoreceptor cells in the retina. The main challenge now is to increase the number of gene therapies in clinical trials and to optimize these treatments for patients, says Dr. Ali in the Commentary "Gene Therapy for Retinal Dystrophies: Twenty Years in the Making."