Sequence 959 (ISIS Cnt1ASO)

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Sequence ISIS Cnt1ASO
Target none
Description Control
Design MOE gapmer
Chemistry moC*moC*moT*moT*moC*C*C*T*G*A*A*G*G*T*T*moC*moC*moT*moC*moC
Sequence moC*moC*moT*moT*moC*C*C*T*G*A*A*G*G*T*T*moC*moC*moT*moC*moC
Application gene silencing
Name ISIS Cnt1ASO

References

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Kordasiewicz HB, Stanek LM, Wancewicz EV, Mazur C, McAlonis MM, Pytel KA, Artates JW, Weiss A, Cheng SH, Shihabuddin LS, Hung G, Bennett CF, Cleveland DW.Neuron. 2012 Jun 21;74(6):1031-44.

Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience. Hache M, Swoboda KJ, Sethna N, Farrow-Gillespie A, Khandji A, Xia S, Bishop KM. J Child Neurol. 2016 Jun;31(7):899-906. PubMed:26823478

Comments

Background

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