Doctoral Candidate - Gene Editing for Cystic Fibrosis


KU Leuven

Job type

Full time

Application deadline

Aug. 15, 2023


Gene Editing for Cystic Fibrosis – Tackling the Translatability Hurdle

This project is focussed on novel gene editing modalities and delivery methods to develop a gene therapy treatment for the monogenetic disease cystic fibrosis which primarily manifests with lung disease.


  • To use gene editing (in particular base and prime editing) for efficient correction of CFTR (gene mutations in CFTR are responsible for cystic fibrosis) in primary human organoid and airway models
  • To develop safe and efficient delivery vehicles for the gene editing machinery, allowing to ultimately target human lung epithelial cells
  • To determine the influence of the (diseased) lung micro-environment in gene editing delivery and editing efficiency

The project will involve wet lab work including the design and cloning of DNA constructs, application of viral vectors (adeno-associated viral vectors) and virus like particles (VLPs), working with primary cells and organ-on-chip systems, immunohistochemistry, microscopy, flow cytometry and other biochemical analyses, and molecular biology including bio-informatic evaluation for gene editing efficiency and safety assessment based on sequencing analysis (Sanger, NGS).

A successful project will result in:

  • A new tool to gene edit CFTR mutations which can effectively and safely target the (human) lung
  • Analysis of the role of the lung microenvironment, in particular inflammation and sputum, which is increased in cystic fibrosis patients, on viral particle delivery
  • Assessment of the role of mucus, inflammation, presence of immune cells, as well as the delivery route, to optimise targeting the desired cells in advanced lung-on-chip models

Enrolment in Doctoral School: KU Leuven

Planned secondments:

  1. INSERM, France (Month 23-24): introduction to the technology platform of virus like particles (‘Nanoblades’)
  2. Universita degli studi di Trento, Italy (Month 30): introduction to novel nucleases for genome editing;
  3. Alia Therapeutics, Italy (Month 31): Introduction to the VesiCas technology platform
  4. Mimetas, Netherlands (Month 36-39): application of developed gene editing and delivery technology to Lung on Chip models

Essential requirements of a successful candidate:

  • You hold a master’s degree in biochemistry, biotechnology, bio-engineering, biomedical sciences, or a related field
  • You are ambitious, well organized, a team player, and have excellent communication skills
  • You are proficient in English both verbal and written *You have the ability to work independently and have a critical mindset
  • You are a pro-active and motivated person, eager to participate in network-wide training events, international travel and public awareness activities

Skills that are viewed as an extra benefit:

  • Solid knowledge of molecular biology and biochemical analyses, and in particular, CRISPR-Cas technology, is an asset
  • Prior experience working with cell culture, and in particular primary cells, possibly in the field of gene therapy, is considered a plus

Contact Person
Marianne Carlon,

LinkProject Description - APPLYLinkMCSA Doctoral Network GET-IN

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