Horizons in Molecular Biology 2017: Tailoring AAV for gene delivery and gene therapy

The developments over the past decade in retinal gene therapy have shown that viral vectors can provide safe gene delivery to the eye’s retina and there is now hope that this treatment option can become a reality in clinical ophthalmology. Subretinal administration of recombinant adeno-associated virus (AAV) has already been demonstrated to be safe and effective in patients with type 2, Leber Congenital Amaurosis, suggesting that AAV-mediated retinal gene therapy may be successfully extended to other blinding conditions in the years to come. This will be possible thanks to the great flexibility of AAV as a vector platform as there are a large number of AAV variants with unique transduction characteristics useful for targeting different cell types in the retina. Cell types that can be transduced using AAVs include glia, epithelium and many types of neurons. Naturally occurring, rationally designed or “artificially evolved” AAV vectors are currently being utilized to target these cell types in the retina and to treat a variety of animal models of retinal disease. The continuous and inventive development of AAV vectors provides opportunities to overcome existing challenges in retinal gene therapy such as transfer of genes exceeding AAV’s cargo capacity, or the targeting of specific cells within the retina or transduction of photoreceptors following minimally invasive intravitreal injections. My talk will describe some of these recent developments in AAV technology, which will make it possible to advance the treatment of a wide range of blinding retinal conditions using gene therapy.

Free registration at www.horizons.uni-goettingen.de