Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models

Abstract Arthrogryposis, Renal dysfunction and Cholestasis (ARC) syndrome is a rare inherited disorder caused by defects in the VPS33B trafficking protein, leading to impaired bile flow, progressive liver disease and early death. No effective treatments are currently available. Gene therapy offers a...

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Detalles Bibliográficos

Principais autores:	Claudiu A. Cozmescu, Mina Nazari, Loukia Touramanidou, Sonam Gurung, Dany Perocheau, Neil Sebire, Yi-Ting Hu, Sian Goldsworthy, Jemima J. Burden, Youssef Khalil, Ivan Doykov, John R. Counsell, Rajvinder Karda, Sergi Castellano, Philippa Mills, Peter Clayton, Wendy Heywood, Dale Moulding, Simon N. Waddington, Julien Baruteau, Giandomenico Turchiano, Paul Gissen
Formato:	Artigo
Idioma:	Inglês
Publicado:	Nature Portfolio 2026-06-01
Series:	Nature Communications
Acceso en liña:	https://doi.org/10.1038/s41467-026-73631-x
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