A new way to advance gene therapy

طريقة جديدة للمضي قدمًا في العلاج الجيني

cover photo human gene therapy. Credit: Dr. Anayi Gonzalez Cordero

A team of scientists from the Children’s Medical Research Institute (CMRI) in Sydney has developed a new way to better target specific organs and tissues in gene therapy – making this innovative gene delivery technology more efficient and described as having “tremendous potential” for the field.

The work was featured in the magazine human gene therapyIt was published this week.

Tools based on adeno-associated virus (AAV), a non-pathogenic virus, are used as a delivery system for gene therapies. To turn the virus into a delivery tool, researchers removed all viruses genes, which turns the virus into a highly effective means of gene delivery, called a vector. The vector can then be used to deliver a working copy of the gene to specific cells in a patient to correct a disease caused by a defective gene. With a single injection, patients can recover from their disease.

However, this system is still in the early stages of development for many conditions. A major limitation is the ability to safely and effectively deliver a therapeutic gene to specific (diseased) cells within the patient’s body; Something the current generation of viral vectors cannot achieve. New bioengineered AAV vectors were developed using Clinical Application Offer a promising alternative in mind. The CMRI team specializes in developing new AAV vectors. The first bioengineering vector to be introduced Clinical Development (Currently in a number of commercial and academic trials, including phase III for the genetic liver condition) It was developed by the CMRI team in collaboration with Stanford University.

In this post, the CMRI team reports a new method that enables the development of more functional and improved AAV vectors. Unlike other commonly used bioengineering methods, the technology developed by the CMRI team allows researchers to select the best new vector, based on how likely it is to restore gene function in a therapeutic application.

“We have successfully improved an existing technique for generating new AAV capsids to be able to target new organs, or target organs that are already accessible. gene therapymore efficiently,” says lead author Adrian Westhaus who is part of CMRI’s Translational Vectorology research unit.

“The optimization was achieved by understanding the current promoter element in the AAV genome and reusing it for engineering the viral capsid.” The promoter is the motor that drives the expression of genes.

It’s an exciting new platform technology, said the Head of the Vector Science Research Unit, Associate Professor Leszek Lisowski.

“This will allow us to develop better new AAVs here in Australia that will enter translational development pathways, with the promise of being better carriers of gene therapies.”

“It will bring hope for effective treatment to millions of children affected by genetic disorders. The technology has tremendous potential, and while its full impact is difficult to predict, it could form the basis for a revolution in biomedicine and translational research.”

Dr. says. Marti Cabanis Creos, inventor of the technology.


Discovery challenges foundations of gene therapy


more information:
Adrian Westhaus et al, AAV-p40 bioengineering platform for variant selection based on Transgene expression, human gene therapy (2022). DOI: 10.1089/hum.2021.278

Provided by the Children’s Medical Research Institute (CMRI)

the quote: A New Way Forward in Gene Therapy (2022, June 15) Retrieved June 15, 2022 from https://medicalxpress.com/news/2022-06-gene-therapy.html

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