14 Sep 2023
Leiden University Medical Center
- Research Field
Medical sciences » Other
- Researcher Profile
First Stage Researcher (R1)
- Application Deadline
29 Feb 2024 - 23:59 (Europe/Amsterdam)
- Type of Contract
- Job Status
- Hours Per Week
- Offer Starting Date
1 Mar 2024
- Is the job funded through the EU Research Framework Programme?
HE / MSCA
- Marie Curie Grant Agreement Number
- Is the Job related to staff position within a Research Infrastructure?
Modern medicine seeks to personalize diagnosis and treatment to the needs of individual patients. This need is heightened in particular for those individual patients where no treatments are available – and where current standard treatment development paradigms exclude to generate such. The ultimate goal of Medicine made to Measure (MMM) is to go even beyond the latest genetic medicine approaches, implementing a novel paradigm of treatment development: the field of single patient tailored antisense oligonucleotide (ASO) treatments for patients with nano-rare disease mutations. Specifically, MMM will develop core modules of a novel “out-of-the-box”, but still EMA-advised treatment development path for these patients - from omics-based theranostics, via preclinical target validation to innovative trial methodology and a pioneering ethical framework for individualised genetic therapies. MMM provides a unique opportunity for doctoral candidates to be involved in building - and to receive training - in all core modules needed for tailored ASO treatment in Europe. This new scientific field requires a new generation of open-minded, technology-fluent and applications-oriented experts.
MMM will help educate future drug development and translational experts in adopting and disseminating such a cross-specialty approach. Our idea for this innovative training network is built on our personal experience and understanding of the importance of single patient tailored genetic treatments in the years to come. We propose a consortium of world-leading experts and partners that present with extended experience and relevant know-how in molecular biology, neurology, translational medicine, biochemistry, bioinformatics, philosophy, mathematics, regulatory science and engineering.
We offer an attractive education and training programme that extends from engagements in high-level research projects, through tailored and network-wide training modules to providing competencies and skill sets that help young researchers go beyond the specific MMM research paradigm. Thus, MMM provides an exceptional platform to young, ambitious and talented researchers who like to engage in supporting the concept of single patient tailored treatments.
PhD PROJECT: Development and efficacy testing of a patient-specific exon skipping ASO
Exon skipping can be applied to skip an exon with a pathogenic variant, provided several criteria are met. In this project, you will design and test antisense oligonucleotides (ASO) to induce skipping of specific exons in genes associated with selected neurodegenerative diseases. You will perform studies to assess whether the resulting proteins are (sufficiently) functional in patient-derived cell cultures (fibroblasts, differentiated IPSCs) and study molecular effects of ASO treatment. For promising ASOs you will perform in vitro safety studies.
- Research Field
- Medical sciences » Other
- Education Level
- Master Degree or equivalent
MSc degree in molecular biology or a related field.
Experience with cell culturing is required. Experience with IPSC culturing and neuronal differentiation is preferred.
Researchers must hold a degree that qualifies them to begin a PhD and be enrolled as a PhD student (usually MA degree or comparable).
Researchers must be doctoral candidates (i.e. not already in possession of a doctoral degree at the date of the recruitment). Researchers who have successfully defended their doctoral thesis but who have not yet formally been awarded the doctoral degree will not be considered eligible.
Researchers must not have resided or carried out their main activity (work, studies, etc.) in the country of the recruiting beneficiary for more than 12 months in the 36 months immediately before their date of recruitment. Compulsory national service, short stays such as holidays, and time spent as part of a procedure for obtaining refugee status under the Geneva Convention are not taken into account.
- Website for additional job details
- Number of offers available
- Leiden University Medical Center
Where to apply
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