Derivation of Large Biorepository of Human iPSC Lines for Open Access Sharing Among Academic Investigators


Pre-Clinical Research

Poster Number: S40


Paul Pang, PhD, Greenstone Biosciences, Christopher Yan, Greenstone Biosciences, Celine Lai, Greenstone Biosciences, Sushma Shenoy, Greenstone Biosciences, Rebecca Yu, Greenstone Biosciences, Zachary Cook, Greenstone Biosciences, Julio Guevara, Stanford University, Yan Zhuge, Stanford University, Joseph Wu, MD, PhD, Stanford University

Background: Human induced pluripotent stem cells (iPSCs) have revolutionized biomedical and translational research in many fields such as neuromuscular diseases, cardiovascular diseases, development, aging, regeneration, cancer, personalized medicine, and many rare genetic diseases. The need for obtaining iPSC lines with the associated donors’ clinical data and from patients of diverse genetic backgrounds has been greater than ever. Greenstone Biosciences Inc. (GSB) has built a curated biorepository to address this demand as an asset for the scientific community utilizing iPSCs.

Methods: The GSB biobank curates clinical data, isolates peripheral blood mononuclear cells (PBMCs), reprograms and banks patient-specific iPSC lines, and performs quality assurance. Reprogramming of PBMCs to iPSCs is carried out using non-integrating Sendai virus encoding for Oct4, Sox2, Klf4, and c-Myc. We then rigorously validate all iPSCs for pluripotency using immunofluorescence of stemness markers, RT-PCR, and SNP-based virtual karyotyping along with quality control methods established as a standard protocol.

Results: We have a growing repository of 2,000 iPSC lines with various disease backgrounds across demographics. We have an equal sex distribution with 53% male and 47% female patients. We have a diverse representation of lines including White (63.6%), Hispanic or Latino (18.8%), Asian (16.2%), and African American (7.0%). Though 13.6% of the repository is from healthy donors, the diseases with genetic mutations include hypertrophic cardiomyopathy (HCM) at 19.8%, dilated cardiomyopathy (DCM) at 15.2%, long QT syndrome (LQTS) at 4.2%, and many other rare conditions such as Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and myotonic dystrophy (DM), which comprise 13.8%. We have also established a simple material transfer agreement (MTA) for open access by academic investigators. Greenstone Biosciences is utilizing this biobank for disease modeling and drug discovery including neuromuscular diseases such as DMD, SMA, and DM1.

Conclusions: We introduce the Greenstone Biosciences iPSC Biobank, a biorepository containing >2,000 human iPSC lines with de-identified patient clinical information and genetic backgrounds. This invaluable resource will help support iPSC research among academic investigators and institutions.