Reviews
Naked DNA Therapy
An article was recently published on ExtremeTech about the use of DNA to cure heart disease. The author does a good job of summarizing the procedure and background of this specific trial. Much of the background is straightforward with a couple of technical terms, but anyone with basic biology knowledge can follow the article easily. His article touches upon all the major steps in gene therapy - modification, delivery, activation, and integration. There is sufficient evidence presented to justify using the SDF-1 DNA to treat heart disease, rather than another gene that codes for cardiac cells. The delivery method is explained clearly as well, with various reasons given to bolster the decision to use naked DNA. However, the reasons for not using a viral vector are quite generic and do not address delivery issues to the heart. The main reason I see in choosing naked DNA over viruses is that naked DNA is safer. Fortunately, the author does mention the downside to naked DNA insertion - low yield or efficiency. Inserting raw DNA into cells can be very ineffective and may end up in more risk than potential payoff. To combat this, the author discusses new technologies which help increase the probability of naked DNA uptake by cells. He also explains the activation of the protein and its function in very general terms. He very quickly mentions integration when discussing the uptake mechanism of SDF-1 DNA into the cells. Generally, there is a balance of pros and cons given by the author, helping to craft an objective view of the procedure. As a result, there is a fairly strong case for the process of this procedure and the reasoning behind it.
Unfortunately, there were quite a few gaping holes in the assessment of the procedure. First, there was not enough background on the patient. All the reader can glean from the article is that the patient has some sort of heart disease. According to Mayo Clinic, heart disease can have many different causes, ranging from atherosclerosis to congenital heart defects. The specific cause of the patient's heart disease is not disclosed, leaving the reader confused about what specifically is being treated. Second, the author does not address what happens to SDF-1 once it enters the heart. Does the SDF-1 create new cardiac tissue? Does SDF-1 generate endothelium cells for blood vessel repair? Is it solely a growth factor? Lastly, the result of the procedure is declared a success without disclosing knowledge of what actually occurred. There is no mention of how the patient's heart had actually been repaired. The author also admits that the effects of SDF-1 and other powerful proteins "are still incompletely known", yet he declares the patient's heart procedure a success. Unless the patient is fully cured on a long term basis and without any side effects, it may be premature to call this a successful procedure. Overall, the article was effective in conveying the point that there is much progress in clinical gene therapy trials to improve patient care.
Unfortunately, there were quite a few gaping holes in the assessment of the procedure. First, there was not enough background on the patient. All the reader can glean from the article is that the patient has some sort of heart disease. According to Mayo Clinic, heart disease can have many different causes, ranging from atherosclerosis to congenital heart defects. The specific cause of the patient's heart disease is not disclosed, leaving the reader confused about what specifically is being treated. Second, the author does not address what happens to SDF-1 once it enters the heart. Does the SDF-1 create new cardiac tissue? Does SDF-1 generate endothelium cells for blood vessel repair? Is it solely a growth factor? Lastly, the result of the procedure is declared a success without disclosing knowledge of what actually occurred. There is no mention of how the patient's heart had actually been repaired. The author also admits that the effects of SDF-1 and other powerful proteins "are still incompletely known", yet he declares the patient's heart procedure a success. Unless the patient is fully cured on a long term basis and without any side effects, it may be premature to call this a successful procedure. Overall, the article was effective in conveying the point that there is much progress in clinical gene therapy trials to improve patient care.
New Genome-Editing Method
MIT Technology Review posted an article about a biotech startup that is redesigning gene therapy targeting. The new method described seems to have great potential for overcoming the hurdle of random gene integration. With a more effective targeting method, it would strongly decrease the possibility of generating secondary diseases, such as cancer, in patients. The author is clearly well versed in biology and is able to effectively articulate the technology utilized by this startup to the general public. There are a couple of links to support the idea and the advantages are discussed at length, with company founders and investors testifying to the novelty of the system. Their treatment would seem to be very effective for single nucleotide polymorphisms, but may be more difficult to implement for mutations that have are the result of multiple genetic mutations. The startup is combating a great obstacle that actually plagued the industry and brought it bad stigma. In 2002, random integration of genes caused four children in gene therapy trials to develop leukemia, casting doubt on the future of gene therapy. So if this company's system is successful, the system may change the future of gene therapy and could accelerate the transition of gene therapy into the medical field.
However, the author fails to address a few issues. First, she does not interview anyone outside of the company. All the quotes and sources are from people who invested in or founded the startup. Those involved with the company will only speak good of the technology, in the hope that more support will be garnered for the project. As a result, a biased and skewed view is given about the potential technology. Without an outside source, there is no way for the reader to craft an objective view regarding the new system of gene targeting. This is only exacerbated by the second issue - there is no reference to the scientific journal that discusses the CRISPR/Cas system. The reader is left to rely upon the author's own interpretation and research regarding the subject, regardless of whether it is accurate or not. The paper would give evidence that has been tested repeatedly and peer reviewed by other scientists in the field. Although the information seems credible, more sources need to be explored before the system could be considered plausible and viable. If respected scientists in the field can attest to the potential of this system, there could be more support and evidence for this venture. Additionally, citing the papers detailing the research that the technology is drawn from will further solidify the reasoning behind this method and may even draw in more investors for the company. Lastly, the author could further expand on the disadvantages of the technology. There are many disadvantages besides the ones she listed and potential solutions that are already in the field could be referenced. Based on what is written in the article, the reader has to put their faith in the company's founders to come up with a novel solution that has troubled the entire gene therapy industry over the past two decades.
However, the author fails to address a few issues. First, she does not interview anyone outside of the company. All the quotes and sources are from people who invested in or founded the startup. Those involved with the company will only speak good of the technology, in the hope that more support will be garnered for the project. As a result, a biased and skewed view is given about the potential technology. Without an outside source, there is no way for the reader to craft an objective view regarding the new system of gene targeting. This is only exacerbated by the second issue - there is no reference to the scientific journal that discusses the CRISPR/Cas system. The reader is left to rely upon the author's own interpretation and research regarding the subject, regardless of whether it is accurate or not. The paper would give evidence that has been tested repeatedly and peer reviewed by other scientists in the field. Although the information seems credible, more sources need to be explored before the system could be considered plausible and viable. If respected scientists in the field can attest to the potential of this system, there could be more support and evidence for this venture. Additionally, citing the papers detailing the research that the technology is drawn from will further solidify the reasoning behind this method and may even draw in more investors for the company. Lastly, the author could further expand on the disadvantages of the technology. There are many disadvantages besides the ones she listed and potential solutions that are already in the field could be referenced. Based on what is written in the article, the reader has to put their faith in the company's founders to come up with a novel solution that has troubled the entire gene therapy industry over the past two decades.