Huntington’s Disease is a hideous progressive disease. One of the primary causes is a mutant protein called mhTT, which is a corrupted essential protein in spinal fluid. mhTT replaces the proper ...

Huntington's disease is caused by an expanded polyglutamine-encoding repeat in the gene huntingtin (HTT) that leads to the accumulation of the mutant HTT protein (mHTT). With the aim of devising ...

Huntington disease (HD) is an incurable, inherited neurological disorder caused by the mutant Huntingtin gene, which produces a mutant form of Huntingtin protein (mHTT). In addition to creating the ...

Several neurodegenerative diseases involve the slow accumulation of a misfolded protein in neurons over many years. The proteins involved in these diseases might differ, but the result is similar — ...

The potential of genome-editing techniques, such as CRISPR/Cas9, to alleviate disease burden has ignited the imagination for thousands of researchers looking for new therapeutic strategies. Scientists ...

A Chinese study led by researchers at Fudan University in Shanghai has described a novel strategy to target the 'undruggable' mutant Huntingtin (mHTT) protein by screening for compounds that directly ...

AMT-130, a gene therapy designed to inhibit the production of mutant huntingtin protein, has shown perplexing results in trials. The safety profile of AMT-130 is acceptable and further data is ...

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed ...

In a new study published in the Brain as cover story, the joint research group of Prof. Xi Chen from Nanjing University and Prof. Zhong Pei from Sun Yat-sen University reports “Therapeutic reversal of ...

CAMBRIDGE, Mass., June 25, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform ...

Mutant huntingtin (mHtt) may now join the ranks of pathogenic proteins that wreak havoc by spreading from one neuron to another. According to a study published July 13 in Nature Neuroscience, mHtt ...

Using time-lapse imaging to follow the path of mutant huntingtin protein from expression through cell death, researchers believe they have laid to rest a much-debated theory of pathogenesis of ...