Gene Therapy uses genes to treat or prevent disease. Types of gene therapy include replacing a mutated gene with a healthy copy, inactivating or knocking out a mutated gene or introducing a new gene to fight disease. In vivo gene therapy approaches directly introduce the gene therapy into the body. Ex vivo approaches in gene therapy first remove cells from the patient and genetic modification occurs outside the body, followed by reintroduction of cells back into the patient.
A CRISPR/Cas9-based strategy, termed homology-independent targeted insertion (HITI) enables targeted gene insertion in non-dividing cells, both in vitro and in vivo. This technique was developed to overcome a barrier to gene therapy in that many technologies are not able to target non-dividing cells such as neurons.
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- CRISPR-Cas9CRISPR-Cas9 is a genome editing system. CRISPR systems provides immunity to bacteria and archaea from viruses and has been adapted for use as a genome editing tool capable of knocking out genes and rewriting genetic sequences in animal, plant and fungi. CRISPR-Cas9 is being adapted to other applications outside genome editing.
- CRISPR/Cas ToolsClustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated (Cas) proteins perform adaptive immune functions in prokaryotic organisms defending against foreign nucleic acids such as viruses. CRISPR/Cas tools have been adapted for use in genome editing and other DNA and RNA targeting applications.