Development of an AAV vector for treatment of Batten disease caused by mutations in TPP1 (CLN2 disease) (360G-Wellcome-109723_Z_15_Z)

£132,626

Dr Sara Mole of University College London is seeking to develop a gene therapy for CLN2 disease. This is one type of Batten disease, an inherited disease that affects young children. The aim is to prevent the Joss of vision that usually occurs as part of this disease as this will improve the quality of life for the children and their families. The work will build on experience that has Jed to clinical trials for other types of blindness, to make a vector that can deliver a healthy copy of the defective gene tothe eye of an animal model with the same disease. The research will test that this is safe and does not cause any problems in the eye. We will/earn more about the effect of the disease on the eye and the effectiveness of this approach at preventing or slowing down the Joss of vision. The results may help to develop gene therapy to treat other symptoms of the Batten disease and other types of disease.

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Grant Details

Amount Awarded 132626
Applicant Surname Mole
Approval Committee Pathfinders Assessment Group
Award Date 2015-08-25T00:00:00+00:00
Financial Year 2014/15
Grant Programme: Title Pathfinder Award
Internal ID 109723/Z/15/Z
Lead Applicant Prof Sara Mole
Partnership Value 132626
Planned Dates: End Date 2018-04-01T00:00:00+00:00
Planned Dates: Start Date 2016-10-01T00:00:00+00:00
Recipient Org: Country United Kingdom
Region Greater London