Sentences with phrase «htt protein»

Wild - type, or healthy, HTT protein is critical for neuronal function, and suppression may have detrimental long - term consequences.
These data indicate that the disease mechanism may at least be partially driven by a loss - of - function in the htt protein.
Knocking down the endogenous htt protein resulted in significant decrease in body weight and hyperactive behavior in the open field.
More research is needed to further explore the role of WT htt in the adult as well as to determine what level of adult WT htt knockdown will be permissible while attempting to reduce the mutant Htt protein.
Current therapeutic strategies for HD are targeted at decreasing htt protein levels but more research is needed prior to advancing these therapies.
Next we these tools to explore how mutations in the HTT protein impact on human cortical development and human neuronal and astroglial functions.
Treatments for Huntington's have typically focused on blocking the activity of the mutant HTT protein, the assumption being that the altered form of the protein was more active than normal, and therefore toxic to neurons.
«Both the role of the HTT protein and the timing of treatment need to be reconsidered; by the time a patient is displaying symptoms, it may be too late to medicate.
However, Brivanlou's work shows that the brain disruption may actually be due to a lack of HTT protein activity.
Next up: a larger trial in hundreds of patients to see if lowering mutant Htt protein slows progression of the disease, then a trial in healthy people who carry the mutant HTT gene to see if antisense treatments could prevent Huntington's altogether.
It halts an intermediate step in the protein - making process by binding to genetic material known as RNA, blocking the issuing of final instructions for making the Htt protein.

Not exact matches

HD is caused by a mutation in the human HTT gene that results in an abnormal expansion and misfolding of the corresponding huntingtin protein.
Mutations of the huntingtin protein (HTT) gene underlie both adult - onset and juvenile forms of Huntington's disease (HD).
The RNAi - mediated silencing of both HTT alleles in neural stem cells derived from hESCs disrupted spindle orientation and led to the mislocalization of dynein, the p150Glued subunit of dynactin and the large nuclear mitotic apparatus (NuMA) protein.
Molecular studies at PsychoGenics and other organizations have demonstrated that the excision of the neomycin resistance cassette results in a modest but significant increase in mutant HTT mRNA levels, and a trend towards an increase in soluble mHTT protein and extranuclear inclusions.
Many functions of huntingtin (htt) protein have been revealed, but the full function is still not completely understood.
The huntingtin (htt) protein has been found to play an essential role during embryogenesis, since knockout Hdh mice do not survive gestation (Duyao et al., 1995; Nasir et al., 1995; Zeitlin et al., 1995).
They zeroed in on the serotonin transporter gene (HTT), which codes for a protein that re-absorbs serotonin into the nerve cell after it has been released.
Individuals carrying the S allele of the 5 - HTTLPR produce significantly less 5 - HTT mRNA and protein, resulting in higher concentrations of serotonin in the synaptic cleft relative to individuals carrying the L allele (Lesch et al. 1996).
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