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Game-changing bacteriophage could revolutionize gene therapy

Ability to carry vast amounts of DNA and other molecules promises substantial advancements in gene and cell therapies

30-May-2023

Key points from article :

A modified version of a bacteriophage, a bacteria-killing virus, has been developed that can deliver substantial amounts of DNA to human cells, paving the way for potential advances in gene therapy. 

This modified phage can transport DNA strands up to 171,000 base pairs long, which is about 20 times the capacity of currently used viruses in gene therapies, according to Venigalla Rao at The Catholic University of America. 

In addition to DNA, this modified virus can also deliver over 1000 other molecules including RNAs and proteins, potentially enabling more comprehensive changes to cells in a single step.

Current gene therapies have faced challenges in treating conditions like Duchenne muscular dystrophy due to the large size of the dystrophin protein gene that exceeds the capacity of existing viruses. The modified virus was able to successfully deliver a full-size dystrophin gene to cultured human cells in lab experiments.

The virus is derived from the T4 bacteriophage, which usually infects specific types of bacteria. The T4 phage has been significantly altered and customized, including an added coating to facilitate uptake by human cells.

The new method could be more cost-effective and easier to produce than current gene therapy delivery systems as they don't need to be grown in human cells.

Further work is needed to prove the viability of the modified virus in delivering genes to cells within the human body. However, it could be immediately applied to modify cells outside the body, potentially enhancing treatments such as cancer immunotherapy.

Research by The Catholic University of America published in Nature Communications.

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Nature Communications

Journal covering all topics in physics, chemistry, and biology.

The Catholic University of America

Private Roman Catholic research university in Washington, D.C.

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Gene Therapy
Game-changing bacteriophage could revolutionize gene therapy