Therapeutic Genomics Centre, DPhil Programme

We are seeking outstanding applicants for 3–4 year, fully funded DPhil/PhD positions at the University of Oxford. These studentships will harness expertise in computational biology and/or translational science to drive the development of cutting-edge therapies to treat rare genetic disorders.

The next round of applications will open in October 2025

The Therapeutic Genomics CoRE

The MRC Centre of Research Excellence in Therapeutic Genomics (MRC CoRE-TG) is a powerful collaboration of over 20 leading laboratories from around the world, including the University of Oxford, Newcastle University, UCL, the University of California (Berkeley and UCSF), and the Karolinska Institute. We also partner with commercial and non-commercial organizations to accelerate our work.

Our mission is to train and empower the next generation of leaders who will shape the future of genomic medicine.

Why rare genetic disorders?

About 5,000 different disorders are caused by a genetic variant at a single location in the genome. Many of these impact a single gene, for example, a mutation to a single base of DNA that impairs the function of the encoded protein. These disorders are often very severe resulting in strong selective pressure so that the variants are not passed on to subsequent generations. Although this selective pressure ensures that the disorders are rare, collectively these disorders affect millions of individuals since there are so many vulnerable genes. With whole-genome sequencing, these specific variants are now routinely diagnosed; the UK is a world leader in the genetic diagnosis through the NHS Genomic Medicine Service (https://www.england.nhs.uk/genomics/nhs-genomic-med-service/) and Genomics England (https://www.genomicsengland.co.uk).

What is genome-targeted therapy?

With recent technological advances, many rare genetic disorders are now potentially treatable. A new class of therapeutic compounds use genetic sequence to target a specific region of DNA or RNA. These include CRISPR-based reagents that can edit DNA and antisense oligonucleotides (ASOs) that modify RNA stability or splicing. These genome-targeted therapies have unrivalled potential for specificity since 16 nucleotides are sufficient to target most of the genome uniquely (4¹⁶ > 3.3 billion); additional nucleotides provide robust genome-wide specificity. Many disease-causing variants can already be targeted and, furthermore, altering just the nucleotide hybridizing sequence can ‘reprogram’ one successful therapy to a new disorder, e.g., by changing just the guide RNA (sgRNA). These therapies could be used as a ‘platform’ to rapidly adapt to treating multiple disorders.

What is Therapeutic Genomics?

To realise the potential of genome-targeted therapies we need to accumulate and share the data and knowledge to predict how changing the targeting sequencing will influence behavior. The term ‘genomics’ describes the use of genome-wide technologies; these, combined with large-scale collaboration and data sharing, have enabled the revolution in the diagnosis of rare disease. ‘Functional genomics’ uses these technologies and principles to add genome-wide insight into gene regulation, including epigenetics and RNA. Therefore, we use the term ‘Therapeutic Genomics’ to describe the application of the principles, approaches, and technologies used in genomics to accelerate the development of effective and safe genome-targeted therapies.

What is the Therapeutic Genomics Centre?

To realize the potential of Therapeutic Genomics, a team of over twenty laboratories are working together to generate the necessary knowledge and experience. These laboratories are based at the University of Oxford, Newcastle University, University College London (UCL), the University of California (including the Innovative Genomics Institute at Berkeley and UCSF), and the Karolinska Institute. In addition, we are assisted by several commercial and non-commercial partners.

How will this benefit your DPhil experience?

Students in the Therapeutic Genomics Centre will receive all the benefits of a DPhil studentship in the Oxford University-MRC Doctoral Training Partnership, including funding, training opportunities, and student wellbeing initiatives (https://www.medsci.ox.ac.uk/study/graduateschool/mrcdtp/training). In addition, students in the Centre benefit from:

• A multidisciplinary team with expertise across the complete range of therapeutic development from target selection and vector delivery to clinical design and regulatory science.
• Personalised training and mentorship: An individual ‘Training Needs Analysis’ will create a customized training plan for you to make the best use of this environment.
• Industry internships: Internships with industry partners will provide trainees with experience of both academic and non-academic career paths.

We are committed to promoting best practices in equality, diversity and inclusivity. We encourage applications from all qualified candidates. 

What projects are available?

There are four fully-funded project proposals via the Oxford-MRC Doctoral Training Partnership portal:

1. Pioneering genomics approaches to develop targeted antisense oligonucleotides for treatment of patients with rare neurological disorders. Supervisors: Prof. Jenny Taylor, Prof. Carlo Rinaldi. Studentship code: MRCCoRETG2025001.

2. Development of antisense oligonucleotide and base editing therapeutics for neurodevelopmental haploinsufficiency disorders. Supervisors: Dr. Thomas Roberts, Prof. Matthew Wood. Studentship code: MRCCoRETG2025002.

3. Developing a Precision RNA Editing Platform for Therapeutic Applications. Supervisors: Prof. Carlo Rinaldi, Prof. Stephan Sanders. Studentship code: MRCCoRETG2025003.

4. Transforming delivery and activity of antisense oligonucleotides (ASOs) outside of the liver with high throughput in vivo ligand screens.  Supervisors: Prof. Matthew Wood, Dr. Alyssa Hill and Dr. Dhanu Gupta. Studentship code: MRCCoRETG2025004.

Ready to join us? Admissions are competitive, and we will select the best applicants for these projects.

General details of how to complete the application form are provided on the ‘How to Apply’ tab of the DPhil in Paediatrics website. Please make sure to include the studentship code for the project(s) you wish to be considered for!

For more information on DPhil in Paediatrics: DPhil in Paediatrics | University of Oxford

For more information about our MRC-Oxford Doctoral Training Programme see: https://www.medsci.ox.ac.uk/study/graduateschool/mrcdtp