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There are currently two clinical trials to correct the deficiency of CLN2 in the brain but there is not study focusing on the loss of vision. The aim of this project is to address this gap by developing a gene therapy treatment for the loss of vision in CLN2 disease.

Who is involved?

The BDFA are delighted to welcome Dr. Mikel Aristorena, who has recently started work in Professor Sara Mole’s lab at the LCMB, UCL. Mikel will divide his time between UCL and the Institute of Ophthalmology (IOP) on a 3-year project funded by the BDFA and the Wellcome Trust.

The work builds on the very successful BDFA-funded project on Gene therapy to treat visual failure in CLN3 disease and Mikel joins Sophia Kleine Holthaus, who was awarded her PhD in January this year. Her BDFA-funded work laid the foundation for this project and this contributed to the successful Wellcome Trust award.

The group at the IOP is headed by Professor Robin Ali and the BDFA would like to thank Dr. Sander Smith and all the members of the team for their continuing support to the BDFA, and the NCL projects.

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