Team:Tsinghua/Project

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Home The Team The Project Parts Submitted to the Registry Modeling Notebook

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Contents

Overall project

Your abstract




Background

Gene Therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one[1]. Although the technology is still in its infancy, it is one of the most promising and active research fields in medicine[1,2]. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods[1].

Vectors in Gene Therapy

Generally, vectors applied in gene therapy can be classified into viral or non-viral.

Viral Methods

All viruses bind to their hosts and introduce their genetic material into the host cell as part of their replication cycle. This genetic material contains basic 'instructions' of how to produce more copies of these viruses, hijacking the body's normal production machinery to serve the needs of the virus[1].

Adenovirus.

Adeno-associated virus.

Retroviruses.

Non-Viral Methods

Non-viral methods present certain advantages over viral methods, with simple large scale production and low host immunogenicity being just two. Previously, low levels of transfection and expression of the gene held non-viral methods at a disadvantage; however, recent advances in vector technology have yielded molecules and techniques with transfection efficiencies similar to those of viruses[1].

Frontiers in Gene Therapy Research

induced Pluripotent Stem Cell (iPS) and Gene Delivery System

Therapeutic microRNA Delivery

Cancer Gene Therapy

Project Details

Introduction

The Experiments

Part 3

Results

References

[1] http://en.wikipedia.org/wiki/Gene_therapy

[2] SM Selkirk. Gene therapy in clinical medicine. Postgraduate Medical Journal.2004;80:560-570; doi:10.1136/pgmj.2003.017764