TY - JOUR
T1 - Generation of an induced pluripotent stem cell line (UCSCi001-A) from a patient with early-onset amyotrophic lateral sclerosis carrying a FUS variant
AU - Martello, Francesco
AU - Lattante, Serena
AU - Doronzio, Paolo Niccolò
AU - Conte, Amelia
AU - Bisogni, Giulia
AU - Orteschi, Daniela
AU - Pirozzi, Filomena
AU - Sabatelli, Mario
AU - Zollino, Marcella
AU - Marangi, Giuseppe
N1 - Funding Information:
This work was supported by grant from the Italian Ministry of Health, Ricerca Finalizzata 2018 (GR-2018-12366086) to GM and by ICOMM (Insieme COntro le Malattie del Motoneurone) ONLUS. We would like to thank the patient and his family for their availability.
Publisher Copyright:
© 2021
PY - 2021/8
Y1 - 2021/8
N2 - Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease affecting upper and lower motor neurons. We generated patient-derived-induced Pluripotent Stem Cells (iPSCs), from an ALS patient affected by an early-onset and aggressive form of the disease, carrying a missense pathogenic variant in FUS gene. We reprogrammed somatic cells using an established Sendai virus protocol and we obtained clones of iPSC. We confirmed their stemness and further generated embryoid bodies, showing their potential of differentiating in all three germ layers. This iPSC line, carrying a pathogenic FUS variant, is a valuable tool to deeply investigate pathogenic mechanisms leading to ALS.
AB - Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease affecting upper and lower motor neurons. We generated patient-derived-induced Pluripotent Stem Cells (iPSCs), from an ALS patient affected by an early-onset and aggressive form of the disease, carrying a missense pathogenic variant in FUS gene. We reprogrammed somatic cells using an established Sendai virus protocol and we obtained clones of iPSC. We confirmed their stemness and further generated embryoid bodies, showing their potential of differentiating in all three germ layers. This iPSC line, carrying a pathogenic FUS variant, is a valuable tool to deeply investigate pathogenic mechanisms leading to ALS.
KW - Amyotrophic lateral sclerosis, FUS, Sendai virus
UR - http://www.scopus.com/inward/record.url?scp=85110662219&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85110662219&partnerID=8YFLogxK
U2 - 10.1016/j.scr.2021.102461
DO - 10.1016/j.scr.2021.102461
M3 - Article
C2 - 34303285
AN - SCOPUS:85110662219
VL - 55
JO - Stem Cell Research
JF - Stem Cell Research
SN - 1873-5061
M1 - 102461
ER -