TY - JOUR
T1 - Delivering a disease-modifying treatment for Huntington's disease
AU - Godinho, Bruno M.D.C.
AU - Malhotra, Meenakshi
AU - O'Driscoll, Caitriona M.
AU - Cryan, John F.
N1 - Publisher Copyright:
© 2014 Elsevier Ltd. All rights reserved.
PY - 2015/1
Y1 - 2015/1
N2 - Huntington's disease (HD) is an incurable genetic neurodegenerative disorder that leads to motor and cognitive decline. It is caused by an expanded polyglutamine tract within the Huntingtin (HTT) gene, which translates into a toxic mutant HTT (muHTT) protein. Although no cure has yet been discovered, novel therapeutic strategies, such as RNA interference (RNAi), antisense oligonucleotides (ASOs), ribozymes, DNA enzymes, and genome-editing approaches, aimed at silencing or repairing the muHTT gene hold great promise. Indeed, several preclinical studies have demonstrated the utility of such strategies to improve HD neuropathology and symptoms. In this review, we critically summarise the main advances and limitations of each gene-silencing technology as an effective therapeutic tool for the treatment of HD.
AB - Huntington's disease (HD) is an incurable genetic neurodegenerative disorder that leads to motor and cognitive decline. It is caused by an expanded polyglutamine tract within the Huntingtin (HTT) gene, which translates into a toxic mutant HTT (muHTT) protein. Although no cure has yet been discovered, novel therapeutic strategies, such as RNA interference (RNAi), antisense oligonucleotides (ASOs), ribozymes, DNA enzymes, and genome-editing approaches, aimed at silencing or repairing the muHTT gene hold great promise. Indeed, several preclinical studies have demonstrated the utility of such strategies to improve HD neuropathology and symptoms. In this review, we critically summarise the main advances and limitations of each gene-silencing technology as an effective therapeutic tool for the treatment of HD.
UR - https://www.scopus.com/pages/publications/84922460332
U2 - 10.1016/j.drudis.2014.09.011
DO - 10.1016/j.drudis.2014.09.011
M3 - Review article
C2 - 25256777
AN - SCOPUS:84922460332
SN - 1359-6446
VL - 20
SP - 50
EP - 64
JO - Drug Discovery Today
JF - Drug Discovery Today
IS - 1
ER -