Design and testing of allele specific gene editing tools. (360G-Wellcome-211705_Z_18_Z)


The Type II Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a programmable RNA guided endonuclease, which is effective at gene editing in mammalian cells. These highly specific and efficient RNA-guided DNA endonucleases may be of therapeutic importance to a range of genetic diseases. The CRISPR/Cas9 system relies on a single catalytic protein, CRISPR associated protein 9 (Cas9), which can be guided to a specific DNA sequence anywhere in the genome by the substitution of a 20-nucleotide sequence, complimentary to the particular target, within a single RNA molecule (sgRNA).A number of computer programs have been developed to predict the sgRNA that cuts the intended target most efficiently with the least off-targetting. I aim to identify, design and characterise sgRNA molecules that could be used to target mutant corneal dystrophy genes by using molecular biology techniques such as in silico identification of mutations in corneal dystrophy genes suitable for CRISPR/Cas9 gene editing with in vitro testing of selected guides, design of sgRNA to target the identified mutation and comparison of sgRNA design and off-target prediction tools.

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Grant Details

Amount Awarded 0
Applicant Surname Boyd
Approval Committee Internal Decision Panel
Award Date 2018-05-31T00:00:00+00:00
Financial Year 2017/18
Grant Programme: Title Vacation Scholarships
Internal ID 211705/Z/18/Z
Lead Applicant Miss Bryony Boyd
Partnership Value 0
Planned Dates: End Date 2018-07-31T00:00:00+00:00
Planned Dates: Start Date 2018-06-01T00:00:00+00:00
Recipient Org: Country United Kingdom
Region Northern Ireland