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Project: Improving the Safety of Genome Editing With Human Kidney Organoids

PI:   Benjamin S Freedman, PhD NIH Report

Genome editing platforms enable the efficient modification of specific DNA sequences, and thus have enormous potential as therapeutics in humans. The kidneys and kidney diseases are important targets for genome editing therapeutics, but there is a dearth of knowledge about how kidneys respond to genome editing, posing a substantial risk of side effects that could do more harm than good. The goal of this project is to use human kidney organoids - 'mini-kidneys' grown in a dish – to develop safe methods to perform genome editing in the kidneys that reduce the risk of inadvertent damage, cancer, and autoimmune disease, as a critical step towards more successful clinical trials.
Summary of data submissions:
  • Data for 1  experiments were submitted on 2024-08-30 SCGE ID:1049

Submissions Details

SCGE ID:1049 - Submission Date: 2024-08-30 
Experiment Name Type Description
Analysis of editing outcomes and adverse events in human kidney organoids

In Vitro Kidney organoids were derived from a human iPS cell line expressing GFP from the safe harbor locus. These were transfected with a commercially available CRISPR ribonucleoprotein complex with guide RNA targeting GFP or a scrambled sequence (control). Editing events were assessed by next generation sequencing of the GFP locus.