Gene and cell based therapies to counteract neuroretinal degeneration  

About the SPP 2127

Vision loss due to inherited eye diseases is a severe burden for millions of patients worldwide. Innovative strategies defeating neuronal dysfunction and cell loss are urgently needed.


The SPP 2127 "Gene and cell based therapies to counteract neuroretinal degeneration" takes centre stage in the attempt to find effective ways to treat rare eye diseases


The retina is neuronal tissue that displays a well-described circuit organization and is easily accessible.


New data generated from this are expected to have tremendous impact on the treatment of other degenerative neuronal disorders and monogenic diseases in general.


Scientists from different research fields (ophthalmology, biology, gene therapy, clinical science, cell based therapies, pharmacology, vectorology, immunology) and a wide range of institutes join forces for an interdisciplinary approach and thus assure networking at its best.


The major challenges arising in this striving field are attributed to four main topics.


Four thematic pillars

Gene Therapy


One therapeutic strategy to target autosomal recessive disorders is referred to as gene addition (or supplementation) therapy, where target cells are provided with a correct cDNA copy.

Cell therapy


Cell therapy strategies aim at adding new cells to the tissue, in this case particularly regarding the replacement of RPE cells and photoreceptors via transplantation.

Development of clinical readout parameter


The SPP 2127 will develop protocols to quantify and compare therapeutic benefits at the retinal and cortical level, to describe results of cell and gene therapy and will develop new read-out parameters.

Factors affecting efficacy of cell and gene therapy


While the current state of experimental therapies is quite advanced, the results of preclinical trials and understanding of pathologies still require work at high levels.

News

By Stieger Knut 16 Apr, 2024
Last week, as a satellite meeting of the famous ProRetina Potsdam meeting, our cientific consortium gathered for a two-day meeting aimed at fostering collaboration and innovation. The event brought us, esteemed professors, principal investigators, and a cohort of bright young scientists and PhD students, once again together - eager to make their mark in the scientific community. The meeting kicked off with a dynamic brainstorming session, as the consortium's budding scientists came together to chart out a unique plan for a patient day. The young scientists and PhD students dove into discussions, sharing ideas on how to engage with patients, understand their needs, and tailor research efforts to address pressing medical challenges. It was inspiring to witness the passion and dedication of these future leaders as they plan a meaningful experience for both patients and researchers alike. Later that day, the PIs and professors joined the meeting for the first scientific talks: the presentations of the young scientists on their newly established collaborative projects, funded by the start up grant of the SPP 2127. After great progress reports, lively discussions and intense scientific exchange, the group transitioned from the conference room to the bowling alley. Laughter and friendly competition filled the air as professors and students alike bonded over strikes and spares, strengthening the bonds that would fuel collaboration in the days ahead. With newfound connections forged and spirits high, day two dawned with a sense of excitement and anticipation. Short talks were on the agenda, providing each PI with the opportunity to showcase their projects and present their ideas for further networking to the consortium. As the meeting drew to a close, thoughts turned to the future. Ideas for networking and ongoing collaboration were exchanged, laying the groundwork for continued innovation and discovery beyond the confines of the meeting room.  In retrospect, the Potsdam consortium meeting was more than just a gathering of minds—it was a celebration of collaboration, curiosity, and the transformative power of science. As the participants dispersed, energized and inspired, they carried with them not only new ideas and connections but a shared commitment to pushing the boundaries of scientific knowledge for the betterment of society.
By Stieger Knut 26 Sep, 2023
In the picturesque town of Weilburg, Germany, where history meets innovation, a remarkable event recently unfolded. Scientists, researchers, and experts from around the world gathered for the annual meeting of the DFG funded research program SPP2127, titled "Gene and Cell-Based Therapies to Counteract Neuroretinal Degeneration." This captivating event, hosted at the Castle Hotel Weilburg, was a melting pot of knowledge, ideas, and collaborations that promises to revolutionize the field of neuroretinal research. SPP2127: Pioneering the Fight Against Neuroretinal Degeneration Neuroretinal degeneration, a group of debilitating eye diseases that cause progressive vision loss, has long been a formidable challenge in the field of ophthalmology. However, thanks to the DFG (Deutsche Forschungsgemeinschaft) funded research program SPP2127, scientists have been granted the resources and platform to pool their talents and tackle this issue head-on. SPP2127 focuses on exploring gene and cell-based therapies to combat neuroretinal degeneration. These innovative approaches hold the promise of not just slowing down the progression of these diseases but potentially reversing the damage, restoring sight to those who have lost it. The Annual Meeting at Castle Hotel Weilburg: A Nexus of Scientific Brilliance The Castle Hotel Weilburg, with its rich history and stunning architecture, provided the perfect backdrop for this momentous scientific gathering. The event unfolded over three days, and each day was filled with intensive discussions, presentations of cutting-edge research, and the sharing of groundbreaking results. The collaborative spirit was palpable as researchers from diverse backgrounds and institutions engaged in spirited debates, shared insights, and brainstormed solutions to the complex challenges posed by neuroretinal degeneration. One of the highlights of the event was the announcement of start-up funding awards for young scientists. These awards not only recognize the potential of these emerging researchers but also invest in the future of neuroretinal research . Congratulations to all the recipients! Exploring Weilburg: A Glimpse into the Past While the primary focus of the meeting was on advancing science, the organizers also ensured that attendees had a chance to explore the charming old town of Weilburg. The second day included a city tour that allowed participants to immerse themselves in the rich history and culture of this beautiful German town. International Collaboration at Its Best SPP2127's annual meeting was not just a local event. It drew invited guests from renowned institutions in Ghent, Basel, Marburg, and the United States, including the National Institutes of Health (NIH). This international presence further enriched the discussions and emphasized the global significance of the research being conducted. Over the three days, the meeting provided a rare opportunity for most members of the consortium and numerous young scientists to come together, connect, and intensify their collaborations. The exchange of ideas, methodologies, and experiences was invaluable, fostering a sense of unity and purpose among all participants. A Bright Future for Neuroretinal Research The annual meeting of SPP2127 at Castle Hotel Weilburg was a testament to the power of collaborative research. As we move forward in the fight against neuroretinal degeneration, the connections made and knowledge shared during this event will undoubtedly pave the way for groundbreaking therapies and, ultimately, the restoration of vision for those in need.  The Castle Hotel Weilburg may have witnessed centuries of history, but the SPP2127 meeting showcased the bright future of neuroretinal research, one where science and collaboration shine as beacons of hope for those affected by these debilitating diseases. As we eagerly await the developments that will emerge from this meeting, we can be certain that the quest to conquer neuroretinal degeneration is stronger and more unified than ever before.
25 Jul, 2023
The city of Dresden, known for its rich cultural heritage and scientific achievements, became a hub of innovation and knowledge during the recent Summer School organized by the Special Priority Programme (SPP2127). This three-day event brought together passionate young scientists, including PhD students and postdocs, to learn, collaborate, and unlock their potential in the field of research. Hosted at the renowned labs of esteemed researchers, Mike O. Karl and Marius Ader, the Summer School provided a unique platform for budding researchers to enhance their grant writing skills, gain valuable insights from fellow students' projects, and embark on their journey to secure internal grants within the SPP2127 framework. Fostering Future Leaders in Research The SPP2127 Summer School is part of the program's commitment to fostering the next generation of scientific leaders. This initiative was designed to empower young scientists by equipping them with essential skills, enabling them to contribute to the growing body of knowledge in their respective fields. The program focused on three key aspects: grant writing, project presentations, and the formulation of proposals for SPP2127 internal grants. Mastering the Art of Grant Writing The Summer School commenced with a series of workshops led by experts in grant writing, where participants gained insights into the nuances of crafting compelling proposals. Seasoned researchers shared their experiences, offering valuable advice on how to identify funding opportunities, structure proposals, and effectively communicate the significance of their research. The young scientists were encouraged to think innovatively, ensuring their projects aligned with the broader goals of SPP2127. Peer Learning and Project Presentations The summer school provided an excellent opportunity for attendees to showcase their ongoing research projects. Each participant presented their work to their peers, fostering an environment of collaboration and constructive feedback. This platform enabled budding scientists to refine their presentation skills and learn from diverse research approaches. The supportive atmosphere encouraged open discussions and stimulated fresh ideas, inspiring participants to tackle research challenges with renewed enthusiasm. The Path to SPP2127 Internal Grants The highlight of the Summer School was the final day, dedicated to present the proposals for SPP2127 internal grants. Armed with the knowledge acquired from grant writing workshops and inspired by peer interactions, the participants pitched their project proposals. Mentors Mike O. Karl and Marius Ader were on hand to provide personalized guidance and expertise. The young scientists were encouraged to explore interdisciplinary collaborations, aligning their projects with the overarching themes of SPP2127. Impact and Beyond The impact of the SPP2127 Summer School extended beyond the three-day event. The participants returned to their home institutions with newfound skills, confidence, and a broader network of collaborators. As they pursued their research projects, they remained connected through an online platform, facilitating continuous support and knowledge exchange.  For many, the Summer School marked the beginning of an exciting journey towards securing internal grants within the SPP2127 program. The scientific community eagerly awaits the outcomes of these proposals, anticipating groundbreaking research that will shape the future of various fields.
By Stieger Knut 05 Apr, 2023
As a research consortium, we recently held a research meeting that brought together PhD students and professors to share their latest findings, exchange ideas, and foster collaborations. Despite the ongoing pandemic, we were able to hold the meeting in person, taking all the necessary precautions to ensure everyone's safety. One of the highlights of the meeting was the science slam for the PhD students. Instead of the traditional poster session, the students had the opportunity to present their research projects in a fun and engaging way, using creative formats such as poetry, music, or theater. The science slam proved to be a fantastic opportunity for the students to showcase their research in a more interactive and dynamic way, engaging the audience in their discoveries and sparking curiosity and interest in their work. The format also allowed for personal and direct feedback from the attendees, as they could ask questions and engage in lively discussions with the presenters. The science slam was not only a great platform for the students to share their research projects, but also a valuable experience in science communication, which is becoming increasingly important in today's world. The students had to think outside the box and find creative ways to present their work, making it accessible and engaging for a wider audience. As a research consortium, our mission is to foster interdisciplinary collaboration, promote innovative research, and contribute to advancing knowledge in our fields of expertise. The research meeting is a key element of our strategy, and we look forward to continuing to organize such events in the future. We believe that by bringing together bright minds and diverse perspectives, we can create a vibrant and dynamic research community that can tackle the most pressing challenges of our times. Overall, the research meeting was a resounding success, with the science slam proving to be one of the most popular and engaging events. It was a great example of how we can foster a culture of innovation, creativity, and collaboration in our research community, by providing opportunities for the next generation of scholars to share their ideas and learn from one another.
By Stieger Knut 17 May, 2022
We are ready for the next chapter!
By Stieger Knut 23 Mar, 2022
T he official confirmation and refusal letters of the DFG were sent out to the applications for the second funding period some weeks ago. We are looking forward to a fresh mix of researchers and topics and can't wait to meet all the new and old PIs for our in-person kick off meeting in June! Still - we are sad that so many great researchers are not a part of the SPP anymore as they did not receive further funding. But we will continue to cooperate and foster our established networks from the first funding period! Until now, the consortium of the second funding period of the SPP 2127 met for a short online meeting to answer the most urgent questions and get to know each other a bit.
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