Deniz Dalkara

Deniz Dalkara is a tenured researcher in INSERM, France and leads a team on gene therapies and animal models of neurodegenerative disease at the Vision Institute in Paris. She graduated from Middle East technical University with a B.S. degree in Biology in 2001. Afterwards, she obtained a masters degree in pharmacology and pharmacochemistry in Strasbourg, France where she later pursued a PhD degree in cellular and molecular aspects of biology. She was awarded the Biovalley PhD thesis for method of protein delivery developed during her graduate studies. Later on she conducted a postdoctoral fellowship in the laboratory of Ernst Babmerg at the Max Planck Institute of Biophysics before moving on to UC Berkeley to do a second post-doctoral training in 2007. At UC Berkeley, Dr Dalkara applied viral engineering principals to enhance AAV vectors for their application in retinal degenerative diseases. Her work includes molecular evolution and engineering of viral gene delivery vehicles and their application to develop innovative gene therapeutic strategies to combat blinding diseases of the retina. For her work in this area, she received Euretina Science and Medicine Innovation award in 2013 and she was selected Innovator under 35 –France by MIT Technology Review in 2014. Dr Dalkara received the Young Investigator award to start her group at the Vision Institute in Paris and has been carrying on her research activities in this research institute with a strong focus on translational research.

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.