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The Latest News on Human Gene TherapiesBY: Josh Ferguson | Category: Industry-News | Submitted: 2013-05-10 03:34:37
Article Summary: "So what is new in the field of gene therapy? Ever since Watson and Crick decoded the mysteries of the human genome in 1953, scientists have been searching for ways to use the wealth of hidden knowledge in order to help those previously thought helpless..."
Ever since Watson and Crick decoded the mysteries of the human genome in 1953, scientists have been searching for ways to use the wealth of hidden knowledge in order to help those previously thought helpless.
The history of gene therapy is marked with beautiful successes and marred by unfortunate failures. The trials for treatment of Severe Combined Immune Deficiency (SCID, or the "bubble boy" syndrome) were halted when some of the children in the trial developed leukaemia like diseases as a direct result of the hematopoietic stem cell vectors used. Conversely, researchers at the National Institute of Health were able to reprogram killer T-cells and have them target metastatic melanoma, successfully. These are only two of the hundreds of trials that have been pushing the boundaries of what we consider "traditional" medicine.
So what is new in the field of gene therapy?
Making a big splash in the UK media, a trial funded by the British Heart Foundation, aims to improve life of those suffering with heart failure. Heart failure is by far and away one of the largest causes of premature death in the UK, with estimates of 82,000 deaths per year, and 2.7 million people suffering from the effects of heart disease. Celladon, a US biotech company, developed a novel treatment, which uses the common cold virus as a vector to inject a gene that encodes the SERCA protein, which will then be expressed within cardiac muscle cells, resulting in larger amounts of calcium, triggering stronger, smoother contractions.
As the SERCA protein can only work within cardiac muscle, there is no chance for unwanted expression in other areas of the human body, which was the cause of the leukaemia cases mentioned above.
Why bother with such technical and complicated treatments? Money. Patients suffering from heart disease can expect to pay tens of thousands of dollars for mechanical heart assistance pumps, or even hundreds of thousands if they require a full heart transplant. This is after decades of expensive and risky medications. If this trial is successful, it will only cost hundreds of dollars and will mean patients do not have to risk dangerous surgeries in order to manage their conditions.
If all goes well, we can expect this kind of radical therapy to be commonplace within a decade.
In order for the bright future of genetic medicine to take place we must be able to quickly genotype and process genetic material from patients in order to best treat them. Doing this used to be an arduous, decade long process that started with the human genome project. However, following the increases in computing power, we are now able to take a sample from a patient and have results within an hour.
Global electronics giant Panasonic has teamed up with Belgian research group, IMEC, to produce a palm sized DNA testing chip.
This revolutionary device is completely automated and is able to produce results from a single drop of blood. It is able to detect at a level of individual single nucleotide polymorphisms, or SNP's, which can be used to identify patients at risk for future illness, whether or not patients will respond well to certain drug regimens.
Traditionally, such results were only obtainable after extensive work in a laboratory, meaning results are obtained after a period of days or even weeks after the sample is taken. IMEC has developed a propriety system that allows them to generate results in a doctors office, in a mere hour.
DNA is extracted from a sample, and the individual regions that require investigation are cut out, then amplified via PCR. The break through process allows for 30 cycles of amplification in a mere 9 minutes.
After that, the sample is filtered with another propriety system, allowing for easy detection of amplified regions, which is then electrochemically identified. Meaning valuable results in hours, all within the palm of your hand.
These two breakthroughs in the field of genetic therapy are just mere tastes of what is slowly becoming possible, as the fields of science and information technology merge.
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