Information für die Wissenschaft Nr. 22 | 9. Mai 2017
Priority Programme „Gene and Cell Based Therapies to Counteract Neuroretinal Degeneration“ (SPP 2127)
The Senate of the Deutsche Forschungsgemeinschaft (DFG, German Research Foundation) has established a new Priority Programme entitled „Gene and Cell Based Therapies to Counteract Neuroretinal Degeneration” (SPP 2127). The programme is designed to run for six years. The present call invites proposals for the first three-year funding period.
Gene and cell based therapies take centre stage in the attempt to find effective ways to treat rare diseases. Vision loss due to inherited retinal dystrophies is a severe burden for two to four million patients worldwide and innovative strategies defeating neuronal dysfunction and cell loss are urgently needed. While the current state of the art is quite advanced in the field of experimental therapies of inherited retinal dystrophies, with clinical trials ongoing for some disorders and functional rescue to a certain level evident, the results of the current trials and preclinical experimentations as well as the improved understanding of the pathology of many inherited retinal dystrophies require continued work at high level. The major points arising in this striving field can be allocated to four main topics that are closely related and intercalate with each other. Proposals within the Priority Programme should address at least one of the following questions:
(A) Gene based therapies:
- How can we improve efficacy of gene addition approaches in order to restore function of target cells in vivo?
- What is the best way to transfer genes into a large area of the retina? How can we realise pan-retinal gene transfer in human patients?
- Is it possible to employ genome editing as a technology to repair disease-causing mutations in vivo?
- Can we sufficiently control cellular DNA repair mechanisms in order to ensure safe modifications to the human genome?
(B) Cell based therapies:
- How can we best obtain, culture and differentiate ipSCs in order to have optimally prepared retinal cells for transplantation?
- Can human ipSC-derived photoreceptors functionally integrate into the degenerated retina?
- Do 3D retinal cultures (i.e. organoids) represent a new potent tool in disease-modelling?
- Can we reliably modify the genome in ipSCs in a way that autologous re-implantation of differentiated retinal cells becomes a safe approach?
(C) Factors influencing the outcome of gene and cell based therapies:
- How does the retina react to subretinal or intravitreal injections of AAV vectors or photoreceptors and/or RPE differentiated from ipSCs?
- Since subretinal injections only treat a defined area of the retina, is the remaining untreated area having an impact (potentially negative) on the treatment outcome?
- How do the major glial cells in the retina, i.e. Müller cells, react to gene or cell therapy and how can we treat them to support therapeutic changes?
(D) Read-out parameters:
- Which are early morphological read-out parameters that allow identifying retinal regions that profit most from a gene or cell based therapy?
- How can we measure functional benefit in treated areas in patients with very low level residual function?
- How to detect reliable function in infants and preverbal children not capable to comply with conventional examination tools?
To foster optimal synergy and collaboration between groups, proposals aimed at treating acquired age related disorders of the retina as well as projects focussing on the clinical description of disorders or addressing mainly genotype-phenotype correlations are excluded from the Priority Programme.
Applications for the first three-year funding period should be written in English and submitted by 30 August 2017 via the electronic elan system, choosing “Schwerpunktprogramm” and selecting “SPP 2127 – Gene and Cell Based Therapies to Counteract Neuroretinal Degeneration” on the menu. The proposals need to follow the guidelines for Priority Programmes (DFG form 50.05) and should be prepared according to the Proposal Preparation Instructions (DFG form 54.01). Furthermore, please send an electronic version (pdf format) of the application to the coordinator (see below).
If you are using the elan system for the first time, please note that you need to register yourself and your institutional addresses before being able to submit a proposal. Also, if you are planning to move to a different institution (e.g. with a temporary position for principal investigators) you need to register the new institutional address beforehand. Please make sure that all applicants of your project start their registration at the latest two weeks before the submission deadline. The registration requests are handled manually by DFG staff.
Please submit the proposal via the electronic system elan:
The DFG forms 50.05 and 54.01 can be found at:
Contact person for questions related to the Priority Programme:
- Prof. Dr. Dr. Knut Stieger,
phone +49 641 985-43835,
Contact person for questions related to the application or review process:
- Dr. Anna Christa,
phone +49 228 885-2632,
Contact person for administrative matters:
- Kim Marita Wind,
phone +49 228 885-2153,
This text is available at www.dfg.de/foerderung/info_wissenschaft/2017/info_wissenschaft_17_22/. Please use this identifier to cite or link to this item.