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

Assoc Prof Imke Tammen Supervisor Profile:
http://sydney.edu.au/vetscience/about/staff/profiles/imke.tammen.php

Assoc Prof Christopher Grupen Supervisor Profile:
http://sydney.edu.au/vetscience/about/staff/profiles/christopher.grupen.php


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).  

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Keywords

Genome editing, animal model, Batten, NCL

Opportunity ID

The opportunity ID for this research opportunity is: 2136