The third dimension: using minibrains to understand brain development changes in HD

The third dimension: using minibrains to understand brain development changes in HD

Researchers show that highly expanded CAGs in the HD gene can cause early developmental changes using 3D brain models called organoids. What’d they find?

Dr Sarah HernandezFebruary 06, 2020

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?

Dr Jeff CarrollJanuary 03, 2020

Could molecular handcuffs lower the protein that causes Huntington's disease?

Could molecular handcuffs lower the protein that causes Huntington's disease?

Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine

Dr Tamara MaiuriNovember 12, 2019

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.

Dr Jeff CarrollAugust 05, 2019

Details emerge of first Huntington’s disease gene therapy clinical trial

Details emerge of first Huntington’s disease gene therapy clinical trial

UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system.

Dr Anna PfalzerJuly 17, 2019

Fresh Updates from First Huntingtin Lowering Study Publication

Fresh Updates from First Huntingtin Lowering Study Publication

Hot off the presses - New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO

Dr Jeff CarrollMay 07, 2019

An Updated Trial Design for Roche's Huntingtin Lowering Trial

An Updated Trial Design for Roche's Huntingtin Lowering Trial

Roche announces an update to the GENERATION HD1 trial after reviewing the ASO open label extension study

Dr Jeff CarrollMarch 21, 2019

When interrupting is good: genetic hiccups that protect against Huntington's disease

When interrupting is good: genetic hiccups that protect against Huntington's disease

Multiple teams find small differences in the 'CAG repeat' bit of the Huntington's disease gene. They don't directly change the huntingtin protein, but do alter the age of symptom onset. What's behind this enigma and what does it mean for patients?

Dr Jeff CarrollMarch 14, 2019

Huntington's disease therapeutics conference 2019 - Day 3

Huntington's disease therapeutics conference 2019 - Day 3

New insights into the huntingtin protein, and further progress in turning genetic quirks that modify the course of HD into treatments, on the final day of the therapeutics conference

Joel Stanton and Dr Jeff CarrollMarch 05, 2019

Huntington's disease therapeutics conference 2019 - Day 2

Huntington's disease therapeutics conference 2019 - Day 2

New tools to bridge the gap between the lab and patients in our update from day 2 of the 2019 HD Therapeutics conference

Joel Stanton and Dr Jeff CarrollFebruary 28, 2019

Huntington's disease therapeutics conference 2019 - Day 1

Huntington's disease therapeutics conference 2019 - Day 1

HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs

Joel StantonFebruary 27, 2019

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