FTD

Broad brain biodistribution conferred by an AAV to restore TDP-43 function mitigates Frontotemporal Dementia-like deficits

Demonstrated that AAV-delivered chimeric TDP-43 repressor (CTR) achieves broad brain biodistribution, restores TDP-43 splicing function, rescues neurons, and mitigates FTD-like …

Tianyu Cao (曹天雨)

• Jan 1, 2025 • 1 min read

TDP-43

Validation of TDP-43 Splicing Repression as a Therapeutic Strategy for ALS and FTD

Conference abstract presented at ASGCT 2024 on validating TDP-43 splicing repression as a therapeutic strategy for ALS and FTD.

Tianyu Cao (曹天雨)

• Apr 1, 2024 • 1 min read

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FTD

Broad brain biodistribution conferred by an AAV to restore TDP-43 function mitigates Frontotemporal Dementia-like deficits

Validation of TDP-43 Splicing Repression as a Therapeutic Strategy for ALS and FTD