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How many is too many? Exploring the toxic CAG threshold in the Huntingtonās disease brain
New work from researchers in London uses mice to narrow in on the number of CAG repeats needed to cause symptoms of Huntingtonās disease. Their work points to fewer than 185 CAGs as a threshold.
By Dr Chris Kay -
Cry your eyes out: detecting huntingtin in tears
Is someone cutting onions? Expanded huntingtin can now be detected in tears to help scientists track disease progression.
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The directorās cut: how CAG repeats change the editing of genetic messages
Scientists in Massachusetts have recently advanced our understanding of how repetitive sequences in DNA can disrupt the creation and editing of genetic messenger molecules in cells, and how this could lead to the production of harmful proteins.
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Understanding expansions at the single cell level
Scientists have looked at CAG expansions in brains from people with HD to see which cells are affected
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Huntington's Disease Therapeutics Conference 2024 – Day 3
HDBuzz is back for the last day of the CHDI HD Therapeutics Conference: Thursday February 29th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. From genes to medicines The morning session will focus on how human genetics is driving the development of therapeutics. āGenetic modifiersā are genes…
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Huntington's Disease Therapeutics Conference 2024 – Day 2
HDBuzz is back for Day 2 of the CHDI HD Therapeutics Conference: Wednesday February 28th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. Itās a brain disease This morningās session is titled āItās a brain diseaseā and will feature talks about BRAINSSSS! HD scientists are a bit…
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Huntington's Disease Therapeutics Conference 2024 – Day 1
Check out research updates from Day 1 of the 2024 HD Therapeutics Conference #HDTC2024
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CRISPR-based drugs: one giant leap for mankind
Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and itās paving the way for similar drugs targeting other diseases. Is Huntingtonās disease next?
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Putting it in print: GENERATION HD1 study results published
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and whatās next?
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Regulating repetition: Gaining control of CAG repeats could slow progression of Huntingtonās disease
Many diseases are caused by repetitive DNA sequences. Understanding the regulation of those repetitive sequences may hold the key for unlocking therapeutics for Huntingtonās disease. A team from Toronto has just advanced our understanding.