Current Status of Stem Cells and Gene Therapy in Ophthalmology

May 3, 2013

In the past couple of months, I was asked to update an article I wrote on stem cells in ophthalmology, originally published in Retina Today, for its sister publication, Advanced Ocular Care, and to write a similar article about the current status of gene therapy for another ophthalmic publication, Retinal Physician. These two articles have now been published in the respective journals and made available online.

Here is a brief summary of each article, along with the link to its online version and a note about finding the current versions of the tables associated with each, online.

The Current Status of Stem Cells in Eye Care, Advanced Ocular Care, March 2013

As noted, this is an update of the original article that appeared in the May/June issue of Retina Today.

“From an inauspicious start several years ago, the use of stem cells in the treatment of several ocular and retinal diseases has picked up steam over the past year.”

The article goes on to describe what stem cells are, the applications of stem cells in the various parts of the eye, a brief discussion of the status of some of the clinical trials, and concludes with a quote from Dr. Stephen Rose, chief research officer of the Foundation Fighting Blindness, who wrote, “Of course, it would be nice if all parts of our bodies, including our retinas, came with extended warranties so you could just swap them out when they go bad. But now that I think about it, that’s what stem cells might do for us someday.”

The Current Status of the Use of Gene Therapy in Ophthalmology, Retinal Physician, April 2013

“With the first approval of a gene therapy treatment for treating a genetic disorder in the Western world, the future of gene therapy for treating ocular disorders looks bright.”

The article goes on to discuss what gene therapy is and how it works; the applications of gene therapy in ophthalmology and clinical trial status for four ocular diseases – Leber’s Congenital Amaurosis, wet AMD, Stargardt Disease, and Usher Syndrom 1b; attempts to answer some remaining questions; and concludes with a quote from officials with the Office of Cellular, Tissue and Gene Therapies (OCTGT) for the FDA, “The recent history of gene therapy has been a mixture of promise and disappointment … Despite the setbacks of the past, the OCTGT shares the enthusiasm of the field and is confident that ongoing clinical investigations will lead to commercially available gene therapy products that are safe and effective and advance the public health.”

In addition, because of the lag between submission and publication of the above articles, the tables that are linked to the print and online versions of the above articles are currently out-of-date. I constantly update their contents and publish the latest versions online, which are accessible from my blog entry about each set of tables.

To access the online versions of the new articles and the updated stem cell and gene therapy tables, please follow this link.




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