Generation of a sheep model of Batten disease using the CRISPR/Cas9 genome editing system

Summary

The neuronal ceroid lipofuscinoses (NCLs, Batten disease) are a group of fatal neurodegenerative inherited diseases in humans and animals (http://bdsra.org/). There is no cure, but trials in animal models and preliminary clinical trials are conducted for some variants of the disease. Large animal models have been instrumental to further the understanding of the genetic basis of the disease and the underlying disease mechanism, and are crucial for safety and proof of concept studies for therapeutic interventions. However, naturally occurring models are not available for all variants and we have received BDSRA funding to use CRISPR/Cas9 technology to create a sheep model for the CLN7 variant of the disease.  

Supervisor(s)

Associate Professor Imke Tammen, Associate Professor Christopher Gerald Grupen

Research Location

Sydney School of Veterinary Science

Program Type

PHD

Synopsis

The research team has experience in animal genetics and advanced reproductive technologies in livestock and is part of an international team of researchers conducting research on Batten disease (BARN, Batten Animal Research Network) The aims of the research/PhD project are: Aim1: Construct development and optimisation of gene editing conditions in primary cell lines and in vitro produced ovine zygotes Aim 2: Genome editing of in vivo produced ovine zygotes Aim 3: Transfer of gene edited blastocysts to recipients to produce CLN7 founder animals Aim 4: Validation of on-target effect and evaluation of off-target activities Aim 5: Characterisation of CLN7 disease in sheep

Additional Information

The successful candidate must have completed an undergraduate degree in the life sciences (e.g. animal science, genetics and reproduction) and have excellent communication skills.  The project will be based at the Camden campus and will provide an excellent postgraduate student training opportunity to gain skills in reproductive biotechnologies and molecular genetics, which will provide for great future career opportunities. In addition, the successful candidate will be supported by an experienced team and well equipped laboratory.   The successful applicant must apply for and be awarded a scholarship (stipend) for example, an Australian Postgraduate Award (APA)) or IPA (International Postgraduate Award). For international students, the student must have a scholarship which covers full tuition fees and a living allowance (http://sydney.edu.au/scholarships/research/postgraduate_awards.shtml).   

The successful candidate will be encouraged to apply for the Post Graduate Research Top-Up Scholarship for Batten Disease Research offered by the Australian chapter of the BDSRA (http://www.battens.org.au/sites/default/files/Australian%20BDSRA%20Top-up%202016%20Scholarship.pdf). 


HDR Inherent Requirements

In addition to the academic requirements set out in the Science Postgraduate Handbook, you may be required to satisfy a number of inherent requirements to complete this degree. Example of inherent requirement may include:

- Confidential disclosure and registration of a disability that may hinder your performance in your degree;
- Confidential disclosure of a pre-existing or current medical condition that may hinder your performance in your degree (e.g. heart disease, pace-maker, significant immune suppression, diabetes, vertigo, etc.);
- Ability to perform independently and/or with minimal supervision;
- Ability to undertake certain physical tasks (e.g. heavy lifting);
- Ability to undertake observatory, sensory and communication tasks;
- Ability to spend time at remote sites (e.g. One Tree Island, Narrabri and Camden);
- Ability to work in confined spaces or at heights;
- Ability to operate heavy machinery (e.g. farming equipment);
- Hold or acquire an Australian driver’s licence;
- Hold a current scuba diving license;
- Hold a current Working with Children Check;
- Meet initial and ongoing immunisation requirements (e.g. Q-Fever, Vaccinia virus, Hepatitis, etc.)

You must consult with your nominated supervisor regarding any identified inherent requirements before completing your application.

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Keywords

Genome editing, animal model, Batten, NCL

Opportunity ID

The opportunity ID for this research opportunity is: 2136