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Open nowPosted 28 hours ago

MSCA-DN PhD in lipid nanoparticle gene therapy for leukodystrophies (London, UK)

EURES job mobility portal314,718 open roles

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Groot-Amsterdam, Netherlands
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Your applicationOpen nowMSCA-DN PhD in lipid nanoparticle gene therapy for leukodystrophies (London, UK)EURES job mobility portal · Groot-Amsterdam, Netherlands

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This job: posted 28 hours ago

The posting

Would you like to contribute to the development of new gene therapies for rare neurological diseases? Work on lipid nanoparticle-based therapies and help bring promising approaches closer to clinical application. POLARIS doctoral network POLARIS is a European Marie Skłodowska-Curie Doctoral Network focused on accelerating therapy development for leukodystrophies: rare genetic disorders affecting the brain's white matter. The network aims to strengthen the full translational pathway, from understanding disease mechanisms and developing disease models to preclinical therapy testing, clinical readiness and patient-informed treatment priorities. To achieve this, POLARIS brings together expertise in leukodystrophy biology, advanced disease models, therapeutic innovation, biomarker development, clinical translation, patient engagement, and ethical, regulatory and health-economic aspects of therapy development. The network will train a cohort of doctoral candidates in an international and interdisciplinary environment, combining academic research, industry-based preclinical development, secondments, network-wide training and transferable-skills development. PhD project For this position, University College London (UCL) is the recruiting organisation. You will be employed by UCL and embedded in the POLARIS consortium. Your academic supervision and disease-specific expertise will be provided by Professor Ahad A. Rahim (UCL School of Pharmacy), Professor Stephen Hart and Dr Julien Baruteau (UCL Great Ormond Street Institute of Child Health). Your PhD project focuses on the in vitro and in vivo preclinical assessment of genetic therapies for leukodystrophies. You will investigate lipid nanoparticle (LNP)-based approaches for delivering therapeutic nucleic acids, such as mRNA, in cellular and animal models. The research follows a translational pathway towards clinical application. You will investigate the functionality, biodistribution and cellular tropism of LNP-mRNA deliver

systems and evaluate their therapeutic efficacy in mouse models of leukodystrophies. A planned secondment at the University of York will provide experience of working with patient-family organisations and exposure to additional disease models. As a doctoral candidate, you will work across different stages of the preclinical research process. Your activities include: Testing the functionality of LNP-mRNA delivery systems in vitro. Analysing the biodistribution of delivery systems in the brain and peripheral tissues and investigating cellular tropism following different administration routes in mice. Conducting a preclinical study using the most promising LNP-mRNA delivery system and administration route in mouse models of leukodystrophies. Evaluating therapeutic efficacy using neuropathology, lifespan, locomotor and behavioural studies. Working with translational research supervisors and POLARIS partners to move promising approaches towards clinical application. We are looking for a candidate with a Master's degree in life sciences and relevant research experience. You bring the following: Experience with molecular technologies, neuroscience and in vivo mouse studies, gained through internships, thesis work or research projects. A strong interest in translational neuroscience, rare disease research and the development of genetic therapies. Experience with LNPs and animal models of neurological disease is an advantage. An accurate and reproducible approach to research, with strong attention to detail and the ability to plan and organise complex experimental work. Good analytical, communication and collaboration skills and the ability to work effectively in an interdisciplinary and international environment. Fluency in spoken and written English is essential for this international research environment. Eligibility criteria Important, strict eligibility criteria according to MARIE SKLODOWSKA CURIE programs apply to applicants: You must not already hold a doctoral

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