Features | Partner Sites | Information | LinkXpress
Sign In
GLOBETECH PUBLISHING
PZ HTL SA
GLOBETECH PUBLISHING

Computer Models Help to Develop Improved Gene Therapy Vectors

By BiotechDaily International staff writers
Posted on 29 Aug 2013
Image: Electron micrograph of adeno-associated viruses (AAVs) (Photo courtesy of Dr. Graham Beards at en.wikipedia).
Image: Electron micrograph of adeno-associated viruses (AAVs) (Photo courtesy of Dr. Graham Beards at en.wikipedia).
Investigators in the field of gene therapy have used an advanced computer algorithm to predict how different adeno-associated viruses (AAVs) can be combined to form the optimum carriers of therapeutic genetic material.

AAVs are small viruses that infect humans and some other primate species. AAVs are not currently known to cause disease, and the virus induces a very mild immune response. Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell. These features make AAV an attractive candidate for creating viral vectors for gene therapy.

To develop quantitative design principles for guiding site-directed recombination of AAV capsids, investigators at Rice University (Houston, TX, USA) examined how capsid structural perturbations predicted by the SCHEMA algorithm correlated with experimental measurements of disruption in 17 chimeric capsid proteins.

They reported in the July 31, 2013, online addition of journal ACS Synthetic Biology that in a chimera population created by recombining AAV serotypes two and four, protection of viral genomes and cellular transduction were inversely related to calculated disruption of the capsid structure. The investigators did not observe a correlation between genome packaging and calculated structural disruption; a majority of the chimeric capsid proteins formed at least partially assembled capsids and more than half-packaged genomes, including those with the highest SCHEMA disruption. Thus, the SCHEMA algorithm should be useful for delineating quantitative design principles to guide the creation of libraries enriched in genome-protecting virus nanoparticles that can effectively transduce cells.

“Gene therapy shows promise in the treatment of not only genetic disorders but also cancer and cardiovascular diseases,” said senior author Dr. Junghae Suh, assistant professor of bioengineering at Rice University. “But you need a mechanism to get the correct gene into the human body and to the target cells. To do that, people use gene vectors, and viruses encompass the largest category of vectors. They have naturally evolved to deliver genes into the body. Our goal is to reprogram them to target specific organs or tissues.”

“The big challenge is to go about this in a rational manner,” said Dr. Suh. “People have done a lot of work to solve the structure of viruses. We know what they look like. The question is: How can we use that information to guide the design of our viral vectors?”

Related Links:
Rice University


Channels

Drug Discovery

view channel
Image: Chitosan is derived from the shells of shrimp and other sea crustaceans, including Alaskan pink shrimp, pictured here (Photo courtesy of NOAA - [US] National Oceanic and Atmospheric Administration).

Chitosan Treatment Clears the Way for Antibiotics to Eliminate Recurrent Urinary Tract Infections

Recurrent urinary tract infection was successfully resolved in a mouse model by treatment with the exfoliant chitosan followed by a round of antibiotics. Bacterial urinary tract infection (UTI), most... Read more

Biochemistry

view channel

Mitochondrial Cause of Aging Can Be Reversed

Researchers have found a cause of aging in lab animals that can be reversed, possibly providing an avenue for new treatments for age-related diseases including type 2 diabetes, cancer, muscle wasting, and inflammatory diseases. The researchers plan to begin human trials late 2014. The study, which was published December... Read more

Therapeutics

view channel

Cytokine Identified That Causes Mucositis in Cancer Therapy Patients

The action of the cytokine interleukin 1-beta (IL-1beta) has been found to underlie the onset of mucositis, a common, severe side effect of chemotherapy and irradiation of cancer patients. Mucositis occurs as a result of cell death in reaction to chemo- or radiotherapy. The mucosal lining of the mouth becomes thin, may... Read more

Business

view channel

Analytical Sciences Trade Fair Declared a Rousing Success

Organizers of this year's 24th "analytica" biosciences trade fair have reported significant increases in both the number of visitors and exhibitors compared to the 2012 event. The analytica trade fair for laboratory technology, analysis, and biotechnology has been held at the Munich (Germany) Trade Fair Center every... Read more
 
Copyright © 2000-2014 Globetech Media. All rights reserved.