ArticleMethods in molecular biology (Clifton, N.J.)2026
Generation and Characterization of Patient-Derived Human Pluripotent Stem Cell Lines for Retinal Gene Therapy.
Article in Methods in molecular biology (Clifton, N.J.), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Patient-derived human-induced pluripotent stem cells (hiPSCs) are a valuable tool for validation of retinal gene therapies. These cells exhibit features identical to human embryonic stem cells (hESCs), such as self-renewal and pluripotency, and can differentiate into various retinal cell types, making them ideal for assessing clinically compliant viral vectors in a human retinal context. Several reprogramming methods have been developed, with integration-free methods being preferable due to their low risk of genomic integration and suitability for clinical applications. This chapter details the generation and characterization of patient-derived hiPSC lines from peripheral blood mononuclear cells (PBMCs) using non-integrative Sendai virus, with a focus on the thorough quality control (QC) necessary post-reprogramming. The QC includes confirming the expression of undifferentiated stem cell markers, the ability to differentiate into the three germ layers, and the absence of chromosomal abnormalities. This protocol provides a stepwise procedure for generating hiPSCs using an integration-free approach in 4 weeks.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.