JOM KITA KE POLITEKNIK

Potential disease modifying therapies for Huntington's disease, lessons learned and future opportunities (Record no. 1609)

MARC details
042 ## - AUTHENTICATION CODE
Authentication code dc
100 10 - MAIN ENTRY--PERSONAL NAME
Personal name Tabrizi, Sarah J
Relator term author
9 (RLIN) 1227
245 00 - TITLE STATEMENT
Title Potential disease modifying therapies for Huntington's disease, lessons learned and future opportunities
260 ## - PUBLICATION, DISTRIBUTION, ETC.
Date of publication, distribution, etc. 2022-07-01.
500 ## - GENERAL NOTE
General note /pmc/articles/PMC7613206/
500 ## - GENERAL NOTE
General note /pubmed/35716694
520 ## - SUMMARY, ETC.
Summary, etc. Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder, for which we have no approved disease-modifying treatments. The molecular pathogenesis of Huntington's disease is complex, with toxicity arising from full length expanded huntingtin and N-terminal fragments of huntingtin, which are both prone to misfolding due to proteolysis, from aberrant intron-1 splicing, and from somatic expansion of the CAG repeat in the HTT gene. Potential therapies for Huntington's disease include those targeting huntingtin DNA and RNA, clearance of huntingtin protein, DNA repair pathways, and other treatment strategies targeting inflammation and cell replacement. The early termination of the tominersen antisense oligonucleotide trials sent a strong signal that it is timely to reflect on lessons learned, where the field stands now, and our challenges and opportunities for the future.
540 ## - TERMS GOVERNING USE AND REPRODUCTION NOTE
Terms governing use and reproduction
546 ## - LANGUAGE NOTE
Language note en
690 ## - LOCAL SUBJECT ADDED ENTRY--TOPICAL TERM (OCLC, RLIN)
Topical term or geographic name as entry element Article
655 7# - INDEX TERM--GENRE/FORM
Genre/form data or focus term Text
Source of term local
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Estevez-Fraga, Carlos
Relator term author
9 (RLIN) 1228
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name van Roon-Mom, Willeke M.C.
Relator term author
9 (RLIN) 1229
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Flower, Michael D
Relator term author
9 (RLIN) 1230
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Scahill, Rachael I
Relator term author
9 (RLIN) 1231
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Wild, Ed J
Relator term author
9 (RLIN) 1232
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Muñoz-Sanjuan, Ignacio
Relator term author
9 (RLIN) 1233
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Sampaio, Cristina
Relator term author
9 (RLIN) 1234
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Rosser, Anne E
Relator term author
9 (RLIN) 1235
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Leavitt, Blair R.
Relator term author
9 (RLIN) 1236
786 0# - DATA SOURCE ENTRY
Note Lancet Neurol
856 41 - ELECTRONIC LOCATION AND ACCESS
Uniform Resource Identifier <a href="http://dx.doi.org/10.1016/S1474-4422(22)00121-1">http://dx.doi.org/10.1016/S1474-4422(22)00121-1</a>
Public note Connect to this object online.

No items available.