A disease-specific iPS cell resource for studying rare and intractable diseases.

ABSTRACT:

Background

Disease-specific induced pluripotent stem cells (iPSCs) are useful tools for pathological analysis and diagnosis of rare diseases. Given the limited available resources, banking such disease-derived iPSCs and promoting their widespread use would be a promising approach for untangling the mysteries of rare diseases. Herein, we comprehensively established iPSCs from patients with designated intractable diseases in Japan and evaluated their properties to enrich rare disease iPSC resources.

Methods

Patients with designated intractable diseases were recruited for the study and blood samples were collected after written informed consent was obtained from the patients or their guardians. From the obtained samples, iPSCs were established using the episomal method. The est

SUBMITTER: Saito MK 

PROVIDER: S-EPMC10485998 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature