PR-001

  • Chemical Properties
CAS No. LY-3884961 Cat. No. BCP49534
Name PR-001
Synonyms PR001A;LY-3884961;LY 3884961;LY3884961;PR 001;PR001;
Formula M. Wt
  • Biological Activity
Description PR001 is a gene-replacement therapy that uses adeno-associated virus 9 (AAV9) to deliver a functional copy of the GBA1 gene to the brain.
Pathways Ion Channel/Membrane Transporter 
Targets GABA Receptor 

Structure

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