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Developing AAV gene therapy for Motor Neuron Disease

Start date

1st October 2022


1 fully funded 3 year Motor Neurone Disease Association (MNDA) PhD Studentship, Basic & Clinical Neuroscience, IoPPN


Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), causes progressive muscular paralysis and death due to the degeneration of motor neurons in the brain and spinal cord.

We discovered the molecular mechanism of toxic gain of TDP-43 function, which recruited polyribosomes in response to cellular stress in the ALS/FTD model (Lee et al., 2022). Currently, we are exploring molecules that can rescue the damaged neuron by TDP-43 proteinopathy for ALS.

Progranulin (PGRN) is a secreted glycoprotein that has important trophic effects on neurons, inhibits inflammation and plays a crucial role in Lysosome. We will exploit PGRN to protect against neuronal death and further develop molecular tools for gene therapy.

The student will investigate the role of PGRN at the synapse using neurons derived from transgenic mice and human stem cells (iPSCs) and acquire cutting-edge cell culture, molecular biology and pre-clinical study techniques.

The project will join one of the world’s leading ALS/FTD gene therapy laboratories based in the Maurice Wohl Clinical Neuroscience Institute. 


Dr Younbok Lee & Professor Chris Shaw

Entry requirements

Applicants should have a Bachelors degree with 2:1 honours. A 2:2 degree may be considered only where applicants also offer a Masters with Merit.

Award types and eligibility

Students will be fully funded for three years full time to include: an annual stipend, fees at the Home rate and travel costs. The studentship is not available to Overseas applicants.

To be treated as a Home student, candidates must meet be/have one of:

  • A UK national (meeting residency requirements)
  • Settled status
  • Pre-settled status (meeting residency requirements)
  • Indefinite leave to remain or enter

Further information advert:

About the IoPPN (link to

Studying at the IoPPN (link to

MSc programmes at the IoPPN (link to

Research degrees at the IoPPN (link to

How to apply

Applicants must complete and submit an online admissions application, via the admissions portal by midnight (23:59 GMT), Friday 5th August 2022. 

On the ‘Choosing a programme’ page, please select Clinical Neuroscience Research MPhil/PhD (Full-time).

In your application, you will be asked to include:

  •  Academic Transcripts – where applicable, academic transcripts must be submitted with the online admissions application
  • Details of your qualifications (you will need to attach copies)
  • Details of previous employment - please include your CV
  • A personal statement describing your interests and why you wish to apply for this project. Please include this as an attachment rather than using the text box.
  • Academic References – all admissions applications require one supporting reference. If the applicant is relying on thier referees to submit a reference directly to the College after they have submitted thier admissions application, then the applicant must ensure that (1) their chosen referee is made aware of the funding deadline (i.e. 7 days from application deadline) and (2) that the reference needs to be sent from an institutional email address.

In the Funding section, please tick box 5 and include the following reference: MNDA-YLCS-22

Please note there is no need to complete the Research Proposal section in your application as the project has already been set.

You are welcome to email (1st supervisor) for more information regarding the project and studentship.

If you have any queries regarding the application process, please contact the Education support team at

References must be received by the deadline for the applicant to be eligible.

Only shortlisted applicants will be contacted.

Closing date

Friday 5th August 2022 (23:59 GMT)


Tuesday 30th August 2022


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