Novel Gene Delivery System Increases Transgene Expression Up to 100-Fold

2017-02-02 11:13:04 by T4N
Health Pat McAfee 

Various retroviruses with distinct advantages for gene delivery have been modified and used in clinical trials. Novel Gene Delivery System Increases Transgene Expression Up to 100-Fold Advertisement Advanced engineering of a mini-intronic plasmid (MIP) system designed to carry a therapeutic gene can significantly enhance the expression of the transgene delivered using an adeno-associated viral (AAV) vector. ‘Advanced engineering of a mini-intronic plasmid (MIP) system designed to carry a therapeutic gene can significantly enhance the expression of the transgene delivered using an adeno-associated viral (AAV) vector by up to 40 to 100-fold.’ Advertisement The ability to increase transgene expression by up to 40 to 100-fold, which would reduce the cost of manufacturing and perhaps also lessen the immune response of AAV/MIP-based gene therapy, is reported in Human Gene Therapy . Authors Jiamiao Lu, Feijie Zhang, and Mark Kay, Stanford University, Palo Alto, CA, and James Williams and Jeremy Luke, Nature Technology Corp., Lincoln, NE, describe the modified MIP expression system in the article entitled "A 5' Non-coding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo." The researchers discuss the potential implications of enhanced transgene expression on the doses needed to achieve a therapeutic response and the flexibility the small intronic sequences offer, allowing them to be used in both DNA plasmids and viral delivery vectors. "Careful observation of the expression characteristics of different vector designs sometimes leads to unexpected findings," says Editor-in-Chief Terence R. Flotte, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "In this case, the authors found that a very substantial increase in the amount of transgene expression (up to 100-fold) could be achieved from rAAV vectors by including essential bacterial plasmid elements in an upstream intron. This could present substantial advantages for future in vivo gene therapy." Source: Eurekalert Advertisement Post your Comments Comments should be on the topic and should not be abusive. The editorial team reserves the right to review and moderate the comments posted on the site. * Your comment can be maximum of 2500 characters Notify me when reply is posted I agree to the terms and conditions Your comments are automatically posted once they are submitted. All comments are however constantly reviewed for spam and irrelevant material (such as product or personal advertisements, email addresses, telephone numbers and website address). Such insertions do not conform to our policy and 'Terms of Use' and are either deleted or edited and republished.Please keep your comments brief and relevant.This section may also have questions seeking help. If you have the information you are welcome to respond, but please ensure that the information so provided is genuine and not misleading. Share

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