Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy.

Kagiava, Alexia; Karaiskos, Christos; Lapathitis, George; Heslegrave, Amanda; Sargiannidou, Irene; Zetterberg, Henrik; Bosch, Assumpció; Kleopa, Kleopas A

Kagiava, Alexia; Karaiskos, Christos; Lapathitis, George; Heslegrave, Amanda; Sargiannidou, Irene; Zetterberg, Henrik; Bosch, Assumpció; Kleopa, Kleopas A.

Afiliação

Kagiava A; Neuroscience Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, 2371 Nicosia, Cyprus.
Karaiskos C; Neuroscience Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, 2371 Nicosia, Cyprus.
Lapathitis G; Neuroscience Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, 2371 Nicosia, Cyprus.
Heslegrave A; Department of Neurodegenerative Disease, UCL Institute of Neurology, London WC1E 6BT, UK.
Sargiannidou I; UK Dementia Research Institute at UCL, London WC1E 6BT, UK.
Zetterberg H; Neuroscience Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, 2371 Nicosia, Cyprus.
Bosch A; Department of Neurodegenerative Disease, UCL Institute of Neurology, London WC1E 6BT, UK.
Kleopa KA; UK Dementia Research Institute at UCL, London WC1E 6BT, UK.

Mol Ther Methods Clin Dev ; 30: 377-393, 2023 Sep 14.

Article em En | MEDLINE | ID: mdl-37645436

Palavras-chave

AAV9; Connexin 32; Golgi-retained mutations; N175D; R75W; X-linked Charcot-Marie-Tooth; demyelination; gene therapy

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Chipre

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google