Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.
Gagnon
Keith T
KT
Department of Pharmacology, UT Southwestern Medical Center, ND8.136B, Dallas, Texas 75390-9041, United States.
Pendergraff
Hannah M
HM
Deleavey
Glen F
GF
Swayze
Eric E
EE
Potier
Pierre
P
Randolph
John
J
Roesch
Eric B
EB
Chattopadhyaya
Jyoti
J
Damha
Masad J
MJ
Bennett
C Frank
CF
Montaillier
Christophe
C
Lemaitre
Marc
M
Corey
David R
DR
D000483:Alleles; D002460:Cell Line; D015870:Gene Expression / Q000187:drug effects; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000188:drug therapy / Q000235:genetics; D009419:Nerve Tissue Proteins / Q000096:biosynthesis* / Q000235:genetics*; D009687:Nuclear Proteins / Q000096:biosynthesis* / Q000235:genetics*; D009841:Oligonucleotides / Q000235:genetics / Q000494:pharmacology; D016376:Oligonucleotides, Antisense / Q000235:genetics / Q000494:pharmacology* / Q000627:therapeutic use; D054735:Phosphorothioate Oligonucleotides / Q000494:pharmacology; D016914:Ribonuclease H / Q000378:metabolism; D013329:Structure-Activity Relationship; D019680:Trinucleotide Repeat Expansion / Q000187:drug effects
C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D009841:Oligonucleotides; D016376:Oligonucleotides, Antisense; D054735:Phosphorothioate Oligonucleotides; C477371:locked nucleic acid; D016914:Ribonuclease H