Generation of two spinal muscular atrophy (SMA) type I patient-derived induced pluripotent stem cell (iPSC) lines and two SMA type II patient-derived iPSC lines

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletion or mutation in SMN1 gene. SMA human induced pluripotent stem cells (iPSCs) represent a useful and valid model for the study of the disorder, as they provide in vitro the target cells. We generated iPSCs from a SMA type I patient and SMA type II patient by using non-integrating episomal plasmid vectors. The resulting iPSCs are episomal-free, express pluripotency markers, display a normal karyotype, retain the mutation (homozygous deletion of SMN1) and are able to differentiate into the three germ layers

Generation of rat-induced pluripotent stem cells: reprogramming and culture medium

The rat represents an animal model highly attractive for studying pharmacology, physiology, aging, cardiovascular diseases, etc., that in many aspects is more adequate than the mouse model. Derivation of induced pluripotent stem cells from rats (riPS) opens the opportunity for gene targeting in specific rat strains, as well as for the development of new protocols for the treatment of different degenerative diseases. Here we report an improved protocol for riPS cell generation, which is based on lentivirus delivery of reprogramming factors with their subsequent excision from the genome, application of serum-free media and chemical inhibitors MEK and GSK. We compared various conditions for riPS cell derivation, analyzed the cell karyotype, and assessed the pluripotency of the established cells. These data may prompt further iPS cell-based gene targeting in rat, as well as the development of iPS-based cell therapy, using this animal model