Each year, BDSRA invites proposals from over 200 scientists working in the field to fund the most promising basic and translational (moving to clinical trial) research. Each proposal is peer-reviewed by experts in lysosomal diseases at universities, children’s hospitals and in the pharmaceutical industry to provide well-rounded reviews.
We are honored to partner with the following organizations who funded this year’s awardees: BDSRA-Australia, BDSRA-Canada, Drew’s Hope, and Noah’s Hope/Hope4Bridget.
The 2017 BDSRA Research Award Winners
Dr. Anthony Cook from the University of Tasmania working in CLN2/CLN3 on a project entitled CRISPR/Cas gene editing of Batten disease genes in patient-specific stem cells.
Dr. Joshua Dearborn from Washington University in St. Louis working in CLN1 on a project entitled Cannabidiol for the treatment of intractable seizures in infantile Batten disease.
Dr. Nadia Mitchell at the University of Otago working in CLN5 CLN6 on a project entitled Towards human translation: Technical support for developing gene therapy for ovine models of CLN5 and CLN6 Batten disease.
Dr. Michel Roberge from the University of British Columbia working in CLN1, CLN2 on a project entitled Therapeutic potential of new premature termination codon readthrough compounds for CLN1 and CLN2 disease.
Dr. Rebecca Whiting from The Curators of the University of Missouri working in CLN2 on a project entitled Sustained TPP1 enzyme delivery for the treatment of CLN2 disease using genetically modified autologous stem cells.