Symposien Freitag

Änderungen im Programmablauf vorbehalten.

Raum Paris 09:45 - 11:15 02.10.2015
Symposium Fr14
Achromatopsia - On the eve of the first ophthalmic gene therapeutic trial in Germany – Joint Symposium of ISCEV and DOG
Vorsitzende/r: Mathias Seeliger (Tübingen), Ulrich Kellner (Siegburg)

The first clinical application of gene therapy in an ophthalmic disorder in Germany, a phase I trial in Achromatopsia, is scheduled to start in 2015. This symposium focuses on the translational steps from the clinical disease via the development of an experimental therapy to the final surgical approach, and will include insights from active trials world-wide and the view of affected patients.
Referent/in: Ulrich Kellner (Siegburg)
Achromatopsia includes a heterogeneous group of disorders characterized by congenital cone dysfunction. Clinical presentation may be variable, and though often believed to be stationary, may include a slowly progressive course of disease. Clinical findings and current diagnostic strategies will be discussed.
Referent/in: Mathias Seeliger (Tübingen)
Achromatopsia is a hereditary, autosomal recessive disorder characterized by the complete unresponsiveness of cones leading to a lack of color discrimination, very poor visual acuity, pendular nystagmus, and photophobia. This presentation provides an overview of the experimental work on gene replacement therapy in achromatopsia from the discovery of rescue effects to the translation to clinical trials.
Referent/in: Dominik Fischer (Tübingen)
Referent/in: Robert MacLaren (Oxford)
Retinal gene therapy requires the precise delivery of viral vector into the subretinal space. A modified surgical technique was developed in the choroideremia gene therapy trials to overcome the challenges of detaching the thin atrophic retina. The positive results of retinal gene therapy seen at 6 months show a sustained treatment effect at 2 years.
Referent/in: Stephen H. Tsang (New York)
Using induced pluripotent stem cells (iPSCs) created from patients allow testing the efficacy of gene or drug-based therapies, elucidating previously unknown mechanisms and pathways of disease, and as a source of autologous cells for transplantation.
Kursreferent: Christina Fasser (Zürich)
There is a great unmet need for therapies in inherited retinal degenerative diseases. Expectations are high, but moderate in outcome: Slowing down the progression of the disease would already been seen as an excellent outcome. Challenges are the missing routine genetic diagnosis and probably in the future the market access of therapies as well as their reimbursement.