May 3, 2018 · Huntington’s disease (HD) is characterized by preferential loss of the medium spiny neurons in the striatum. Using CRISPR/Cas9 and somatic nuclear transfer technology, we established a knockin (KI) pig model of HD that endogenously expresses full-length mutant huntingtin (HTT).

Jul 9, 2018 · Huntington’s disease is a severe neurodegenerative monogenetic disease that results in age-dependent selective neuronal loss in affected individuals—a feature that cannot be mimicked in...

Mar 28, 2023 · They applied the AAVs to the striatum of HD KI pigs by stereotaxic injection, and the injected AAVs successfully “hunt out” the CAG repeat expansion with remarkably high efficiency and corrected it with a normal 20×CAG by homology directed repair (HDR) following CRISPR-mediated DNA cleavage.

This chapter will focus on the important findings from large animal models of Huntington’s disease and discusses the use of large animal models to investigate the pathogenesis of Huntington’s disease and develop new therapeutic strategies.

Feb 21, 2023 · In a pig model of HD, a single brain injection of a viral vector carrying the gene-editing molecules cut the amount of mutant protein in half, and improved the animal’s gait months later. The strategy deserves further study, the authors suggest.

Mar 29, 2018 · A team of scientists has established a pig model of Huntington's disease (HD), an inherited neurodegenerative disease, anticipateing that the pigs could be a practical way to test treatments for HD.

Feb 16, 2023 · A Cas9-based gene therapy that replaces expanded CAG repeats in the mutant HTT allele causing Huntington’s disease by a normal CAG repeat led to substantial reductions in neurological symptoms...

Huntington’s disease is a severe neurodegenerative monogenetic disease that results in age-dependent selective neuronal loss in affected individuals—a feature that cannot be mimicked in...

Nov 14, 2018 · In the latest example of the benefits of using swine tissue as models in biomedical research, scientists have engineered the world’s first pigs with Huntington’s disease, which could lead to a cure for this debilitating illness.

Huntington’s disease (HD) is characterized by the preferential loss of the medium spiny neurons in the striatum. Using CRISPR-Cas9 and somatic nuclear transfer technology, we established a knock-in (KI) pig model of HD that endogenously expresses full-length mutant huntingtin (HTT).