New Technology Makes Gene Therapy More Effective

November 10, 1998

Better Weapon to Fight Liver Disease, Sickle Cell Anemia, Other Genetic Diseases

CHICAGO, November 10 -- Research that could point the way to more effective gene therapies for liver and other diseases was made public at The American Association for the Study of Liver Diseases 1998 Annual Meeting in Chicago, November 6 - 10.

Betsy T. Kren, PhD, of the University of Minnesota Medical School reported on a study of a new technology that permanently corrects genetic defects caused by what are called "single point mutations" in the liver cells of laboratory rats. Most gene therapy uses viruses to deliver altered bits of DNA to specific genes to correct defective DNA. But viruses have limited effectiveness, in part because they deliver DNA randomly to the genes. In the study, researchers used a new technology to replace the bad DNA in liver cells with new healthy DNA much more effectively and efficiently.

According to Clifford Steer, MD, head of the project, researchers put altered bits of DNA into specially designed targeting molecules and injected the molecules into tail veins of rats. Those bits of DNA made their way to the nuclei of genetically damaged liver cells. Through a complicated process, the DNA bits "tricked" the damaged cells into precisely correcting the specific defective nucleotide in the DNA that the researchers had targeted.

"The potential future impact of this technology is profound," says Steer. "Most genetic diseases of the liver are single point mutations, as are sickle cell and many other non-liver diseases."
-end-
AASLD is the leading medical organization for liver researchers and physicians. Founded by physicians in 1950, AASLD's mission is to advance the science and practice of hepatology. Today, AASLD provides representation and education for more than 2,200 hepatologists worldwide.



American Association For The Study of Liver Diseases

Related DNA Articles from Brightsurf:

A new twist on DNA origami
A team* of scientists from ASU and Shanghai Jiao Tong University (SJTU) led by Hao Yan, ASU's Milton Glick Professor in the School of Molecular Sciences, and director of the ASU Biodesign Institute's Center for Molecular Design and Biomimetics, has just announced the creation of a new type of meta-DNA structures that will open up the fields of optoelectronics (including information storage and encryption) as well as synthetic biology.

Solving a DNA mystery
''A watched pot never boils,'' as the saying goes, but that was not the case for UC Santa Barbara researchers watching a ''pot'' of liquids formed from DNA.

Junk DNA might be really, really useful for biocomputing
When you don't understand how things work, it's not unusual to think of them as just plain old junk.

Designing DNA from scratch: Engineering the functions of micrometer-sized DNA droplets
Scientists at Tokyo Institute of Technology (Tokyo Tech) have constructed ''DNA droplets'' comprising designed DNA nanostructures.

Does DNA in the water tell us how many fish are there?
Researchers have developed a new non-invasive method to count individual fish by measuring the concentration of environmental DNA in the water, which could be applied for quantitative monitoring of aquatic ecosystems.

Zigzag DNA
How the cell organizes DNA into tightly packed chromosomes. Nature publication by Delft University of Technology and EMBL Heidelberg.

Scientists now know what DNA's chaperone looks like
Researchers have discovered the structure of the FACT protein -- a mysterious protein central to the functioning of DNA.

DNA is like everything else: it's not what you have, but how you use it
A new paradigm for reading out genetic information in DNA is described by Dr.

A new spin on DNA
For decades, researchers have chased ways to study biological machines.

From face to DNA: New method aims to improve match between DNA sample and face database
Predicting what someone's face looks like based on a DNA sample remains a hard nut to crack for science.

Read More: DNA News and DNA Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.