Generation of four patient-specific pluripotent induced stem cell lines from two Brazilian patients with amyotrophic lateral sclerosis and two healthy subjects

• Published in: Stem cell research Vol. 37; p. 101448
• Main Authors: Gubert, Fernanda, Vasques, Juliana F., Cozendey, Tatiane D., Domizi, Pablo, Toledo, María Fernanda, Kasai-Brunswick, Tais H., Loureiro, Marli P.S., Lima, José M.B., Gress, Claudio H., Cabello, Giselda M.K., Cabello, Pedro H., Borgonovo, Tamara, Vaz, Isadora M., Silva, Rosane, Mendez-Otero, Rosalia
• Format: Journal Article
• Language: English
• Published: England Elsevier B.V 01.05.2019; Elsevier
• Subjects: Adult; Amyotrophic Lateral Sclerosis - genetics; Amyotrophic Lateral Sclerosis - pathology; Cell Differentiation; Cells, Cultured; Cellular Reprogramming; Healthy Volunteers; Heterozygote; Humans; Induced Pluripotent Stem Cells - metabolism; Induced Pluripotent Stem Cells - pathology; Leukocytes, Mononuclear - metabolism; Leukocytes, Mononuclear - pathology; Male; Mutation; Phenotype; Vesicular Transport Proteins - genetics
• ISSN: 1873-5061; 1876-7753; 1876-7753
• DOI: 10.1016/j.scr.2019.101448

Induced pluripotent stem cell (iPSC) lines were generated from erythroblasts of two patients with amyotrophic lateral sclerosis (ALS) and two healthy individuals. One familial and one sporadic ALS patients were used, both with genetic alterations in VAPB gene. CytoTune™-iPS 2.0 Sendai Reprogramming Kit (containing the reprogramming factors OCT3/4, KLF4, SOX2 and cMYC) was used to generate the iPSC cell lines. The four iPSCs express pluripotency markers, have normal karyotype and differentiated spontaneously in the three germ layers. The expression of Sendai virus was lost in all iPSC lines after 15 passages.