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Open Access Establishment of a secure differentiation and de-differentiation-inducing method by protein transduction system LENA

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The LENA method for gene therapy promises to be a safer technique for gene editing and cell fate redirection than current methods which can have unwanted effects, including insertional carcinogenesis and carcinogenic overexpression of the delivered gene. Gene therapy is a broad term for a range of methods which modify the genetic code of a host cell with the aim of fighting or preventing disease. Researchers are working on gene therapies to fight many different conditions including immune-deficiencies, Parkinson's Disease and haemophilia. The underlying premise is that a mutated or deficient gene in the patient's cells can be replaced with a properly functioning DNA segment, edited to function normally, or be deleted to prevent it causing future disease. However, gene therapy currently remains a highly promising technique that is still mostly practised in vitro or in animal models, as researchers strive to perfect reliable and consistent methods of delivery without serious side-effects.

For the last three years, Dr Jun Komano, Head of the Clinical Laboratory at Nagoya Medical Centre in Japan, has been working to perfect a method of changing cell DNA, which he hopes will avoid some of the downsides associated with other methods of gene editing. The LENA project achieves protein transduction using a pseudo-typed lentivirus nano-particle, which promises to be a safer method for cell fate redirection and gene-editing. He points out: 'a technology to deliver gene editing in a highly regulated manner, without damaging the host cell genome and predisposing cells to becoming malignant, is necessary for gene therapy to become a practical and effective means of treating disease in the clinic.'
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Document Type: Research Article

Publication date: March 1, 2018

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