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Gene therapy

Gene therapy

Gene Therapy uses genes to treat or prevent disease. Type 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.

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Meredith Hanel
Meredith Hanel edited on 20 Jun 2019 2:41 pm
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Gene therapy

Gene Therapy uses genes to treat or prevent disease. Type 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.

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Gene Therapy uses genes to treat or prevent disease. Type 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 genome editinggene therapy

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Using genome-editing technologies to correct specific mutations represents a potentially transformative new approach for treating genetic disorders. Despite rapid advances in the field of genome editing, it is still unclear whether the long-standing goal of in vivo targeted transgene integration is feasible. 



This is primarily because current tools are inefficient. In particular, current technologies are incapable of targeted gene knock-in in non-dividing cells, the major building blocks of adult tissues. This poses a significant barrier for developing therapeutic strategies to treat a broad range of devastating genetic disorders. 



A unique CRISPR/Cas9CRISPR/Cas9-based strategy, termed homology-independent targeted insertion (HITI)homology-independent targeted insertion (HITI), which enables targeted gene insertion in non-dividing cells, both in vitroin vitro and in vivoin 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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Dawson Sewell
Dawson Sewell edited on 3 May 2019 12:48 pm
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In vivo genome editing

HITI method

Using genome-editing technologies to correct specific mutations represents a potentially transformative new approach for treating genetic disorders. Despite rapid advances in the field of genome editing, it is still unclear whether the long-standing goal of in vivo targeted transgene integration is feasible. 



This is primarily because current tools are inefficient. In particular, current technologies are incapable of targeted gene knock-in in non-dividing cells, the major building blocks of adult tissues. This poses a significant barrier for developing therapeutic strategies to treat a broad range of devastating genetic disorders. 

...

A unique CRISPR/Cas9-based strategy, termed homology-independent targeted insertion (HITI), which enables targeted gene insertion in non-dividing cells, both in vitro and in vivo. 

Dawson Sewell
Dawson Sewell edited on 3 May 2019 12:38 pm
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Dawson Sewell
Dawson Sewell edited on 15 Mar 2019 10:52 am
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 Gene therapy

Gene Therapy uses genes to treat or prevent disease. Type 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.

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