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Gene therapy

Gene therapy

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.

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. 

In vivo gene therapy

HITI method

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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