The Batten Disease Support and Research Association’s 2016 Research Grants have Been Awarded

The Batten Disease Support and Research Association (BDSRA) is pleased to announce its 2016 research grant awardees. BDSRA have partnered with BDSRA-Australia, Drew’s Hope, Noah’s Hope-Hope4Bridget, Beyond Batten Disease Foundation, and the Thisbe and Noah Scott Foundation to co-fund these important projects.

Background: The BDSRA annual research-merit review cycle begins in November of each year. Through a ‘Request for Letters of Intent’ that will be issued later this year, interested researchers are invited to submit a letter of intent (LOI) outlining the research they want to conduct. For the upcoming call for LOIs, BDSRA seeks innovative research projects that have the potential to advance therapeutic strategies for all or any of the neuronal ceroid lipofuscinoses. Each award, depending on funding availability, will be no more than $60,000 for a one-year period. Those candidates receiving the highest scores are asked to offer full proposals, which are then reviewed by a panel of three scientists. The top proposals are forwarded to the BDSRA board for approval and funding. Put the BDSRA on your radar this autumn, if you have some innovative research you are thinking of proposing.

The 2016 BDSRA research grant awardees include:

• Rebecca Whiting, The University of Missouri, “Sustained TPP1 enzyme delivery for the treatment of CLN2 disease using genetically modified autologous stem cells,” $55,000.

• Imke Tammen and Dr. Christopher Grupen, University of Sydney, Australia, “Generation of a sheep model of Batten disease using the CRISPR/Cas9 genome editing system,” $50,000.

• Marco Sardiello, Baylor College of Medicine, “mTOR-independent lysosomal enhancement for the treatment of neuronal ceroid lipofuscinoses,” $30,000.

• Tammy Kielian, The University of Nebraska Medical Center, “Link between ‘danger signals’ and inflammasome activation in the pathogenesis of juvenile Batten disease,” $40,000.

• Stephanie Hughes, University of Otago, New Zealand, “Cross-correction in CLN6 Batten disease,” $40,000.

• Jacob Cain, Sanford Research, “Determining the neuronal specific mechanisms of CLN3 in juvenile neuronal ceroid lipofuscinose,” $45,000.

• Taina Autti, University of Helsinki, Finland, “Advanced diffusion MRI in Batten disease (CLN3): white matter microstructure and brain connectivity,” $50,000.

• Steven Gray, University of North Carolina, Chapel Hill, “INCL gene therapy using AAV9 vectors,” $30,000.

• Jonathan Mink, University of Rochester Medical Center, “BDSRA Registry,” $30,000. Dr. Mink has also been a Lysosomal Disease Network researcher, with his LDN #6717 study entitled “Clinical and Neuropsychological Investigations in Batten Disease.”