The newest of the new in gene therapy: 'Tag and target and exchange'
A combination of two techniques promises to improve the efficiency and effectiveness of experimental gene therapies, while also reducing potential side effects says a new research report published in the December 2011 issue of the FASEB Journal. The report describes how scientists from Germany combined two techniques involving the use of site-specific recombinases, or enzymes that facilitate the exchange of genetic material between DNA strands, to help guide exactly where new genetic material is inserted into a cell's DNA. This experimental approach to gene therapy represents an important advance, as successful gene therapy has the potential to correct the root cause of numerous illnesses and health conditions.
"The central outcome of these and related techniques is the predictability and safety of a therapeutic regimen," said Juergen Bode, a researcher involved in the work from the Institute of Experimental Hematology at Hanover Medical School in Hanover, Germany. "These novel strategies will obviate the majority of animal experiments that are presently needed; it will enhance the effectiveness and shorten the timeline."
To make this discovery, Bode and colleagues identified two types of site-specific recombinases (SSR), one from yeast (Flp recombinase) and one from phages (PhiC31 recombinase), which are capable of tagging and targeting specific areas in a DNA strand. Specifically, the tagging process involves mounting a distinct address within a genome, whereas the targeting process covers the delivery of genetic material to this address. PhiC31was identified as an ideal enzyme for tagging because it recognizes just a limited number of pre-existent genomic addresses with well-known and mostly beneficial characteristics, allows for only a one-way transfer of genetic material, and is basically irreversible. In contrast, Flp recombinase acts in a reversible manner, meaning that a given target can be modified over and over again to study the role of slight changes in the structure of therapeutic proteins. Although both techniques have particular properties, they complement one another and may even be used in conjunction to obtain ultimate results.
"Successful gene therapy has been called the Holy Grail of biomedical research. It could revolutionize medicine as much, if not more, than the development of antibiotics," said Gerald Weissmann, M.D., Editor-in-Chief of the FASEB Journal. "While we have a long way to go, this report is an important advance. It shows us how to insert important genetic material exactly where it is needed, rather than blindly popping it into a cell and hoping for the best."
More information: Soeren Turan andJuergen Bode. Site-specific recombinases: from tag-and-target- to tag-and-exchange-based genomic modifications. FASEB J. December 2011 25:4088-4107; doi:10.1096/fj.11-186940
- New reagents for genomic engineering of mouse models to understand human disease Aug 19, 2009 | not rated yet | 0
- Finding the ZIP-code for gene therapy: Scientists imitate viruses to deliver therapeutic genes Aug 31, 2009 | not rated yet | 0
- Insect virus holds the key to safer stem cell therapy Jul 29, 2011 | not rated yet | 0
- Scientists find new agent to fight genetic disorders -- Zorro-Locked Nucleic Acid Apr 30, 2007 | not rated yet | 0
- The promise of stem cell-based gene therapy Jun 27, 2011 | not rated yet | 0
- Motion perception revisited: High Phi effect challenges established motion perception assumptions Apr 23, 2013 | 3 / 5 (2) | 2
- Anything you can do I can do better: Neuromolecular foundations of the superiority illusion (Update) Apr 02, 2013 | 4.5 / 5 (11) | 5
- The visual system as economist: Neural resource allocation in visual adaptation Mar 30, 2013 | 5 / 5 (2) | 9
- Separate lives: Neuronal and organismal lifespans decoupled Mar 27, 2013 | 4.9 / 5 (8) | 0
- Sizing things up: The evolutionary neurobiology of scale invariance Feb 28, 2013 | 4.8 / 5 (10) | 14
Classical and Quantum Mechanics via Lie algebras
Apr 15, 2011 I'd like to open a discussion thread for version 2 of the draft of my book ''Classical and Quantum Mechanics via Lie algebras'', available online at http://lanl.arxiv.org/abs/0810.1019 , and for the...
- More from Physics Forums - Independent Research
More news stories
Northwestern University scientists have shown a gene involved in neurodegenerative disease also plays a critical role in the proper function of the circadian clock.
Genetics May 16, 2013 | not rated yet | 0 |
Informed consent is the backbone of patient care. Genetic testing has long required patient consent and patients have had a "right not to know" the results. However, as 21st century medicine now begins to use the tools of ...
Genetics May 16, 2013 | 5 / 5 (1) | 3 |
Ethicists provide framework supporting new recommendations on reporting incidental findings in gene sequencing
In a paper published in Science Express, a group of experts led by bioethicists in the Center for Medical Ethics and Health Policy at Baylor College of Medicine provide a framework for the new American College of Medical Geneti ...
Genetics May 16, 2013 | not rated yet | 0
The use of genome-wide analysis (GWA), where the entirety of an individual's DNA is examined to look for the genomic mutations or variants which can cause health problems is a massively useful technology for diagnosing disease. ...
Genetics May 16, 2013 | not rated yet | 0
DNA databases might help identify victims of crime and human trafficking, but how do we safeguard the personal privacy of innocent victims and family members? A new report online May 15 in the Cell Press journal Trends in ...
Genetics May 15, 2013 | not rated yet | 0
The latest makeover to a massive psychiatric tome honored by some, reviled by others and even called the "Bible" of mental disorders is being released Saturday with a host of new changes.
24 minutes ago | not rated yet | 0
A new case of the deadly coronavirus has been detected in Saudi Arabia where 15 people have already died after contracting it, the health ministry announced on Saturday on its Internet website.
44 minutes ago | not rated yet | 0
An increasing number of U.S. children are experiencing gastrointestinal issues that require interventions to resolve, according to research presented at Digestive Disease Week (DDW).
2 hours ago | not rated yet | 0 |
A ground-breaking advance in colonoscopy technology signals the future of colorectal care, according to research presented today at Digestive Disease Week(DDW). Additional research focuses on optimizing the minimal withdrawal ...
2 hours ago | 5 / 5 (1) | 0
Big names in medicine are set to give an upbeat assessment of the war on AIDS on Tuesday, 30 years after French researchers identified the virus that causes the disease.
11 hours ago | 5 / 5 (1) | 0
For combat veterans suffering from post-traumatic stress disorder, 'fear circuitry' in the brain never rests
Chronic trauma can inflict lasting damage to brain regions associated with fear and anxiety. Previous imaging studies of people with post-traumatic stress disorder, or PTSD, have shown that these brain regions can over-or ...
12 hours ago | 5 / 5 (1) | 0 |