A human CAGinSTEM platform for decoding HTT repeats’ somatic instability links CAG interruption to HD pathology in neurons

Summary: Somatic CAG instability in the mutant Huntingtin (HTT) gene is increasingly recognized as a key hallmark of Huntington’s disease (HD). Using our novel human CAGinSTEM platform, we manipulated cis genetic elements influencing instability in human HD neurons, monitoring repeat length. Quality...

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Principais autores:	Martina Zobel, Gianluca Damaggio, Maria Lidia Mignogna, Dario Besusso, Davide Scalzo, Andrea Cossu, Camilla Trovesi, Mariacristina Crosti, Francesco Cortina, Ilaria Campus, Giulio Formenti, Saveria Mazzara, Francesco Gregoretti, Laura Antonelli, Gennaro Oliva, Chiara Zuccato, Vincenza Colonna, Paola Conforti, Matteo Cereda, Riccardo Lorenzo Rossi, Simone Maestri, Andrea Scolz, Raffale Iennaco, Elena Cattaneo
Formato:	Artigo
Idioma:	Inglês
Publicado:	Elsevier 2025-12-01
Series:	Cell Reports
Assuntos:	CP: Neuroscience
Acceso en liña:	http://www.sciencedirect.com/science/article/pii/S2211124725014573
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