Document is current
Any future updates will be listed below
-
Authors
Pagliari, Elisa
Anastasia, Alessia
Bellandi, Floriana
Garbellini, Manuela
Ongaro, Jessica
Taiana, Michela
Comi, Giacomo P.
Ottoboni, Linda
Sierra-Delgado, Julieth Andrea
Likhite, Shibi
Meyer, Kathrin C.
Nizzardo, Monica
Corti, Stefania P.
-
Funding
Funding for this research was provided by:
Fondazione Cariplo (2015-0776)
Ministero dell’Istruzione, dell’Università e della Ricerca (Dipartimenti di Eccellenza Program 2023–2027)
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (PNRR-POC-2023-12377653)
Ministero della Salute (PNC-E3-2022-23683266)
- License Information
-
More Information
Article History
Received: 20 May 2025
Accepted: 23 November 2025
First Online: 4 January 2026
Declarations
:
: All animal procedures received approval from the Institutional Animal Care and Use Committee (IACUC) of the University of Milan and the Italian Ministry of Health, adhering to national guidelines (D.I. no. 116, G.U. supplement 40, February 18th, 1992; Circular no. 8, G.U., July 14th, 1994) under the approved protocol 707/2019-PR.
: Not applicable.
: M.N, S.C. and K.M. are inventor of the international patent PCT/US20/61863: “AAV Gene Therapy for Spinal Muscular Atrophy with Respiratory Distress Type I (SMARD1; OMIN 604320) and Charcot-Marie-Tooth Disease 2S (CMT2S) Caused by Mutations in the IGHMBP2 Gene”.
