Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases

Abstract excerpt

<title>Abstract</title> <p>Gene correction of multiple alleles for compound heterozygous recessive or polygenic diseases is a promising therapeutic strategy. However, the targeting of multiple alleles using genome editors in a single cell could lead to mixed genotypes and adverse events that amplify during tissue morphogenesis. Here we demonstrate that <italic>Spy</italic>Cas9-based S1mplex genome editors can be...

Topics

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• CRISPR-Cas Systems
• Gene Editing
• Gene Transfer Techniques
• Genetic Therapy
• Glycogen Storage Disease Type II
• Induced Pluripotent Stem Cells
• Inheritance Patterns
• Primary Cell Culture

Identifiers and source

Literature Corpus work

634df701-18f6-5bf8-a323-cd7a0953e9f4

DOI

10.21203/rs.3.rs-90150/v1