A new treatment for ALS

Project specs

Format

2D

Contact

Chris Mueller

Country

USA

Funding agency

Mueller lab and Brown lab at University of Massachusetts Medical School

Length

01:30

Summary

A new therapy approach for C9ORF72 linked ALS: using AAV9 as a vehicle to deliver an artificial microRNA to target aberrant transcripts. A single dose will permanently treat motor neurons and prevent cell death.

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