Novel MeCP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates

Autor: Powers, Samantha, Likhite, Shibi, Gadalla, Kamal K., Miranda, Carlos J., Huffenberger, Amy J., Dennys, Cassandra, Foust, Kevin D., Morales, Pablo, Pierson, Christopher R., Rinaldi, Federica, Perry, Stephanie, Bolon, Brad, Wein, Nicolas, Cobb, Stuart, Kaspar, Brian K., Meyer, Kathrin

Publikováno v: In Molecular Therapy July 2023

PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD

Autor: Pilotto, Federica, Schmitz, Alexander, Maharjan, Niran, Diab, Rim, Odriozola, Adolfo, Tripathi, Priyanka, Yamoah, Alfred, Scheidegger, Olivier, Oestmann, Angelina, Dennys, Cassandra N, Sinha Ray, Shrestha, Rodrigo, Rochelle, Kolb, Stephen, Aronica, Eleonora, Di Santo, Stefano, Widmer, Hans Rudolf, Charlet-Berguerand, Nicolas, Selvaraj, Bhuvaneish T, Chandran, Siddharthan, Meyer, Kathrin, Zuber, Benoît, Goswami, Anand, Weis, Joachim, Saxena, Smita

Publikováno v: Pilotto, F, Schmitz, A, Maharjan, N, Diab, R, Odriozola, A, Tripathi, P, Yamoah, A, Scheidegger, O, Oestmann, A, Dennys, C N, Sinha Ray, S, Rodrigo, R, Kolb, S, Aronica, E, Di Santo, S, Widmer, H R, Charlet-Berguerand, N, Selvaraj, B T, Chandran, S, Meyer, K, Zuber, B, Goswami, A, Weis, J & Saxena, S 2022, ' PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD ', Acta Neuropathologica . https://doi.org/10.1007/s00401-022-02494-5
Pilotto, Federica; Schmitz, Alexander; Maharjan, Niran; Diab, Rim; Odriozola, Adolfo; Tripathi, Priyanka; Yamoah, Alfred; Scheidegger, Olivier; Oestmann, Angelina; Dennys, Cassandra N; Sinha Ray, Shrestha; Rodrigo, Rochelle; Kolb, Stephen; Aronica, Eleonora; Di Santo, Stefano; Widmer, Hans Rudolf; Charlet-Berguerand, Nicolas; Selvaraj, Bhuvaneish T; Chandran, Siddharthan; Meyer, Kathrin; ... (2022). PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD. Acta neuropathologica, 144(5), pp. 939-966. Springer-Verlag 10.1007/s00401-022-02494-5
Pilotto, F, Schmitz, A, Maharjan, N, Diab, R, Odriozola, A, Tripathi, P, Yamoah, A, Scheidegger, O, Oestmann, A, Dennys, C N, Sinha Ray, S, Rodrigo, R, Kolb, S, Aronica, E, di Santo, S, Widmer, H R, Charlet-Berguerand, N, Selvaraj, B T, Chandran, S, Meyer, K, Zuber, B, Goswami, A, Weis, J & Saxena, S 2022, ' PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD ', Acta Neuropathologica, vol. 144, no. 5, pp. 939-966 . https://doi.org/10.1007/s00401-022-02494-5
Acta neuropathologica 144(5), 939-966 (2022). doi:10.1007/s00401-022-02494-5
Acta neuropathologica, 144(5), 939-966. Springer Verlag
Acta Neuropathologica, 144(5), 939-966. Springer Verlag

Acta neuropathologica 144(5), 939-966 (2022). doi:10.1007/s00401-022-02494-5
Published by Springer, Berlin ; Heidelberg

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8d5cf249b50a70ecbaf50f60dd560d99
https://hdl.handle.net/20.500.11820/c3ba6926-b9b1-489d-ad10-d0e576e890e2

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