Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients.

Autor: Signorelli M; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands., Ebrahimpoor M; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands., Veth O; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Hettne K; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Verwey N; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., García-Rodríguez R; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Tanganyika-deWinter CL; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Lopez Hernandez LB; Departamento de Medicina Genómica, Universidad Autónoma de Guadalajara, Guadalajara, Mexico.; Centro Médico Nacional '20 de Noviembre', ISSSTE, Ciudad de México, Mexico., Escobar Cedillo R; Sociedad Mexicana de la Distrofia Muscular A.C INR-LGII, Ciudad de México, Mexico., Gómez Díaz B; Sociedad Mexicana de la Distrofia Muscular A.C INR-LGII, Ciudad de México, Mexico., Magnusson OT; deCODE genetics/Amgen, Reykjavik, Iceland., Mei H; Sequencing Analysis Support Core, Leiden University Medical Center, Leiden, The Netherlands., Tsonaka R; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands., Aartsma-Rus A; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Spitali P; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.

Publikováno v: EMBO molecular medicine [EMBO Mol Med] 2021 Apr 09; Vol. 13 (4), pp. e13328. Date of Electronic Publication: 2021 Mar 10.

Antisense Oligonucleotide Therapy for the Treatment of C9ORF72 ALS/FTD Diseases.

Autor: Riboldi, Giulietta, Zanetta, Chiara, Ranieri, Michela, Nizzardo, Monica, Simone, Chiara, Magri, Francesca, Bresolin, Nereo, Comi, Giacomo, Corti, Stefania

Publikováno v: Molecular Neurobiology; Dec2014, Vol. 50 Issue 3, p721-732, 12p