Template-independent genome editing and repairing correct frameshift disease <i>in vivo</i>

Abstract excerpt

Frameshift mutation caused by small insertions/deletions (indels) often generate truncated and non-functional proteins, which underlies 22% inherited Mendelian disorders in humans. However, there is no efficient in vivo gene therapy strategies available to date, especially in postmitotic systems. Here, we leveraged the non-homologous end joining (NHEJ) mediated non-random editing profiles to compensate the frames...

Topics

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• CRISPR-Cas Systems
• Dependovirus
• Disease Models, Animal
• Extracellular Matrix Proteins
• Gene Editing
• Genetic Therapy
• High-Throughput Nucleotide Sequencing
• Usher Syndromes

Identifiers and source

Literature Corpus work

55348c74-7751-5ea3-a07a-9a4ce0807071

DOI

10.1101/2020.11.13.381160