NEW YORK And PARIS, November 15, 2023 /PRNewswire/ –The PolG Foundation is a nascent foundation established in 2022 by a PolG family, with a mission to support and accelerate research to find effective treatments and a cure for PolG mitochondrial disorders.
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With a dedicated and experienced Board of Directors and Scientific Advisory Board, including some of the world’s leading mitochondrial scientists, the Foundation has quickly become a leading funder of basic, translational and clinical PolG research. In collaboration with the global scientific community, we are investing in the development of essential tools and continuing to make our discoveries available to scientists, clinicians and patients around the world in the field of mitochondria.
In addition to the development of IPS cell lines, RNA sequencing, and two transgenic mouse models for use by PolG researchers, the Foundation is also actively working to create a global PolG registry and prospective study of natural history.
Frederick of Nassau, a POLG warrior and son of the founding parents, designs a MITO clothing line to raise awareness and funds for the PolG Foundation. View the store – https://shopmito.polgfoundation.org/
A central pillar of the Foundation is to raise funds and award grants to world-class researchers in the areas of basic science, clinical trial research, and the development of novel therapies for mitochondrial PolG disorders in populations. Pediatric and adult PolG. Today, the PolG Foundation is pleased to announce four winners awarded to our first call for proposals for research initiatives.
We are excited to see these grants implemented by the four research groups that will pursue exceptional R&D work in the field of POLG biology:
- Professor Vamsi K. Mootha
Expanded Institute of WITH And Harvard, Boston (UNITED STATES)
- Title: A variant-function map of POLG via in-depth mutational analysis
- Synopsis of the work: POLG deficiency is associated with enormous allelic heterogeneity and, at present, it is very difficult to know which variants observed are benign or pathogenic. The goal is to create a comprehensive dashboard for POLG that relates all possible variants to biological and biochemical functions. Next-generation DNA synthesis to create a saturation mutagenesis library for POLG will be combined with functional genetic screens that quantitatively evaluate POLG variants. This will be a durable toolkit that will be valuable to both basic research and clinical communities.
- Professor Anu Suomalainen Wartiovaara
University of HelsinkiResearch Programs Unit, Faculty of Medicine (Finland)
- Title: TargetPOLG: POLG disease mechanisms and therapeutic approaches
- Synopsis of the work: The Finnish cohort of POLG patients we are supporting has the same homozygous mutation, but manifests with symptom variability, indicating that strong environmental and/or genetic factors affect the variable outcomes of POLG disease from childhood to adulthood. ‘middle age. Understanding and targeting these mechanisms have the potential to control POLG disease progression. More specifically, mechanisms will be explored and identified that could contribute to and promote the manifestations of POLG disease. Selected molecular targets will be tested and challenged to generate preclinical evidence for human trials.
- Dr. Kristina Xiao Liang,
Department of Clinical Medicine, University of Bergen (Norway)
- Title: Stem cell-based study for drug discovery for POLG disease
- Synopsis of the work: The overarching goal of the project is to establish a human stem cell-based 2D neural system and a 3D brain organoid platform for drug discovery and to identify mitochondria-targeted therapies that are ready for repurposing and can rapidly trigger clinical trials in POLG patients.
- Dr Yi Shiau Ng,
Wellcome Center for Mitochondrial Research (WCMR), University of Newcastle (UK)
- Title: Coalition for POLG Trial Preparation (C4TR-POLG): Clinical Trial Readiness in POLG-Related Mitochondrial Diseases and Ataxia: A Prospective, Longitudinal Study Identifying Sensitive and Ecologically Valid Biomarkers
- Synopsis of the work: Studying treatments against ultra-rare forms of mitochondrial diseases is a challenge. Participant pools are small and limited by rigid inclusion and exclusion criteria. There is often an incomplete understanding of genotype-phenotype relationships and natural history to inform the development of trial endpoints. Addressing these disparities is recognized as a complex and long-standing conundrum. There is an urgent need to harness the value of innovative multi-stakeholder networks.
Fully aligned with the Foundation’s strategy, these research grants cover different elements that characterize the disease: from understanding its development and progression to monitoring phenotypes with outcome measures. We hereby commit to discovering and developing potential new drugs and therapies.
As the standard bearer of the global PolG community, we are deeply moved by all the interest and support received from individuals, foundations, scientific, biotechnology, pharmaceutical, patient advocacy and medical ecosystems. . With your help and financial support, we can continue our ambitious mission of discovering new therapies and a potential cure for PolG. THANKS!
Make a donation:
https://polgfoundation.kindful.com/
©2023 | The POLG Foundation is a 501(c)(3) nonprofit organization. EIN87-1876876. Donations are tax deductible in the United States, to the extent permitted by law. Donations can be sent to: THE POLG FOUNDATION, 99 Park Ave., 24th Floor, New York, NY 10016.
Contact:
Paula Casanova
paula@polgfoundation.org
SOURCE The PolG Foundation
