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SUMMARY:Gene targeting therapies – what does the future hold for neurolo
 gical disorders? - Professor Sarah Tabrizi\, University College London
DTSTART:20200127T193000Z
DTEND:20200127T210000Z
UID:TALK129019@talks.cam.ac.uk
CONTACT:John Cook
DESCRIPTION:*An overview of antisense oligonucleotide therapies in develop
 ment for neurodegenerative diseases.*\n\nThere are no effective disease mo
 difying therapies for neurodegenerative diseases such as Alzheimer’s (AD
 )\, Parkinson’s (PD)\, amyotrophic lateral sclerosis (ALS) or Huntington
 ’s disease (HD). Huntington’s disease (HD) is a devastating autosomal 
 dominantly inherited neurodegenerative disease and the genetic predictabil
 ity of HD provides an opportunity for early therapeutic intervention many 
 years before overt symptom onset and at a time when reversal or prevention
  of neural dysfunction may still be possible. As HD is monogenetic\, fully
  penetrant\, and characterised by a long premanifest phase\, it is emergin
 g as a potential model for studying therapeutic intervention in other neur
 odegenerative conditions such as Alzheimer’s or Parkinson’s disease wh
 ere no preclinical diagnostic tests exist. In addition\, HD manifests with
  a broad range of clinical symptoms and signs\, many of them common to the
 se other diseases\, and involves widespread pathology throughout most of t
 he brain involving similar protein misfolding. Understanding of HD pathoge
 nesis is evolving\, and I will present an overview of important approaches
  in development for targeting mutant HTT DNA and RNA\, the cause of HD pat
 hogenesis\, and in particular I will present our recent successful phase 1
 b/2a clinical trial testing the effects of antisense oligonucleotide thera
 py (ASO) with RG6042 (formerly known as IONIS HTT Rx) in patients with ear
 ly Huntington’s Disease and present the results of the first successful 
 HTT-lowering drug trial (Tabrizi et al New England Journal of Medicine 201
 9). This study is the first to demonstrate antisense-mediated protein supp
 ression in patients with a neurodegenerative disease. While this particula
 r ASO holds great promise for HD\, our findings have broader implications.
  These data suggest that antisense technology has the potential to provide
  disease-modifying benefits in other neurodegenerative diseases associated
  with aberrant production of proteins\, including ALS\, Alzheimer’s dise
 ase and many other diseases that currently lack adequate treatments. In my
  talk I will review ASO approaches in development for CNS diseases.\n\nAll
  welcome – free for CSAR members\, £2 for undergraduates\, postgraduate
 s and recent PhDs\, £5 for non-members.\n\n"Add to calendar":https://www.
 addevent.com/event/?hB3803314\n\n"Find out more about this event":https://
 www.csar.org.uk/lectures/lectures-2019-2020/huntingtons/\n\n"Find out more
  about CSAR membership":http://www.csar.org.uk/membership/\n
LOCATION:The Wolfson Hall\, Churchill College\, Storey's Way\, Cambridge\,
  CB3 0DS
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