JHU Ref #: C18058
Value Proposition:
- GBA1 knockout SH-SY5Y human neuroblastoma cell line
- Generated using CRISPR/Cas9 KO technology
- Unavailable from any other source
- No observable off-target effects
Unmet Need:
It has been demonstrated that GBA1 deficiency contributes to the aggregation of α-synuclein (α-syn) and the transition from monomeric α-syn to α-syn tetramers/multimers, which is characteristic of Parkinson’s disease, Gaucher’s disease, and other α-synucleinopathies. However, no GBA1 knockout cell lines are currently available from either academic institutions or commercial sources. As a result, researchers lack the tools to study the specific role of GBA1 deficiency in these disease states. This cell line model addresses a critical gap for in vitro experimentation.
Technology Description:
Researchers at Johns Hopkins have developed a human neuroblastoma cell line deficient in glucocerebrosidase 1 (GBA1). This cell line is a valuable tool for studying Parkinson’s disease, Gaucher’s disease, and other α-synucleinopathies. It enables researchers to investigate the effects of GBA1 deficiency in these conditions.
Stage of Development: The cell line is completely generated and ready for sale.
Data Availability: Data available upon request.
Inventor(s): Hanseok Ko, PhD
Publication
- Kim, et al., PNAS (2018): available here
