- Gene Editing for Huntington’s Disease Shows Promise in Pigs
This revealed that, by either administration route, gene editing had created an insertion or deletion in the huntingtin gene that disrupted protein expression, rather than replacing the gene with a healthy version, in about two-thirds of cells
- Cas9-mediated replacement of expanded CAG repeats in a pig . . . - ALZFORUM
While the pathogenesis underlying HD is relatively clear, there are no effective disease-modifying treatments In this study, the authors used an adeno-associated virus (AAV)-mediated CRISPR Cas9 system to replace the expanded CAG repeats and lower the expression of mHTT protein in a huntingtin knock-in pig model
- Could Taking Down a DNA Repair Protein Stave Off Huntington’s?
At birth, people who carry a string of more than 40 CAG repeats within the first exon of the huntingtin gene are all but destined to develop Huntington’s disease Yet, recent studies are converging on the idea that the number of inherited repeats is not always sufficient to manifest the disease
- Prion-like properties of the mutant huntingtin protein in living . . .
Alpaugh M, Denis HL, Cicchetti F Prion-like properties of the mutant huntingtin protein in living organisms: the evidence and the relevance Mol Psychiatry 2022 Jan;27 (1):269-280
- TRIM37 is a primate-specific E3 ligase for Huntingtin and accounts for . . .
Qin Y, Chen L, Zhu W, Song J, Lin J, Li Y, Zhang J, Song X, Xing T, Guo T, Duan X, Zhang Y, Ruan E, Wang Q, Li B, Yang W, Yin P, Yan XX, Li S, Li XJ, Yang S TRIM37 is a primate-specific E3 ligase for Huntingtin and accounts for the striatal degeneration in Huntington's disease Sci Adv 2024 May 17;10 (20):eadl2036 PubMed Recommends Please login to recommend the paper
- Huntington’s Protein Charged with Trans-synaptic Spread of Pathology
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 aggregates pass from neurons in the cortex to neurons in the striatum via synaptic vesicles The
- Huntingtin (-poly) | ALZFORUM
Specificity Huntingtin Comments Questions No Available Comments Make a comment or submit a question To make a comment you must login or register References Paper Citations Peters PJ, Ning K, Palacios F, Boshans RL, Kazantsev A, Thompson LM, Woodman B, Bates GP, D'Souza-Schorey C Arfaptin 2 regulates the aggregation of mutant huntingtin protein
- Huntingtin, BDNF, Neurodegeneration: Is Speed of the Essence?
In the July 10 Cell, Frederic Saudou, Sandrine Humbert, and colleagues from the CNRS in Orsay, France, report that huntingtin (htt) enhances transport of brain-derived neurotrophic factor (BDNF) along microtubules The results offer a plausible explanation as to why striatal neurons, which depend on
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