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AAV-CAMKII-CofilinS3A
- Development stage
- Preclinical
- Lead developer
- University of Pennsylvania
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
AAV-CAMKII-CofilinS3A is a viral gene therapy construct designed for research purposes to express a constitutively active form of cofilin (CofilinS3A) specifically in excitatory neurons. The construct utilizes an adeno-associated virus (AAV) vector and the CaMKII (calcium/calmodulin-dependent protein kinase II) promoter to drive expression. The S3A mutation (Serine to Alanine at position 3) prevents the inhibitory phosphorylation of cofilin, resulting in a protein that is persistently active in regulating actin dynamics. In preclinical studies, particularly those involving sleep disruption and Alzheimer's disease models, this tool is used to investigate how activated cofilin contributes to synaptic loss, neural injury, and impaired calcium clearance.
- Other names
- AAV9-CaMKII-CofilinS3AAAV-9-CaMKII-CofilinS3AAAV 9-CaMKII-CofilinS3AAAV-CaMKII-Cofilin(S3A)
02
Targets
CFL1 (Cofilin-1)
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