Project Details
Description
Since we now have identified phenotypes in neuronal models of CLN7 Batten’s disease,
here we aim to test the efficacy of novel therapeutics that are customized to correct the unique
mutations expressed by the patients. In collaboration with Dr. Timothy Yu, we will determine if
novel antisense oligonucleotide drugs can restore lysosomal function, reduce pathological
storage material, and halt neurodegeneration in vitro. In addition to neuronal models we will
characterize the function of glial cell cultures of CLN7 patients. Recent studies have indicated that
astrocyte dysfunction and neuroinflammation may precede and contribute to neuron loss in
Batten’s disease [4]. Therefore, we will assess their function and contribution to neuron
dysfunction in vitro. Our studies will impact the field by discovering novel therapies, as well as
determine the contribution of glial cell dysfunction in CLN7 Batten’s disease.
Status | Finished |
---|---|
Effective start/end date | 3/1/22 → 2/29/24 |
Funding
- Mila's Miracle Foundation, Inc. (MMF AGMT 3-30-22)
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