In lieu of an interview transcript, I have decided to summarize the interviews I conducted. All the ideas summarized below are Dr. Schwob's responses to my interview questions.
Dr. James Schwob is a professor of Developmental, Molecular and Chemical Biology at the Tufts University Sackler School of Graduate Biomedical Sciences. I began my interview with Dr. Schwob by asking if the public should have any misgivings about the CRISPR/Cas9 technology and editing the human germline and where it’s heading. Dr. Schwob noted that there are potential concerns since CRISPR/Cas9 is a powerful technology that could modify the genome of children and has the potential to be harmful or wrong. He noted that the applications of the technology could extend to be like those presented in the novel Brave New World, where embryos are grown in factories and one could easily modify the development of embryos that could harm the embryo or the individual. Another concern that Dr. Schwob mentioned was that of proper regulation and benefits outweighing the risks
When asked about whether we should use CRISPR/Cas9 to change human genes in ways that can be passed down to future generations, Dr. Schwob stated that there is nothing inherent in the technology that would make it wrong to use is that way. For instance, we admit the possibility of allowing therapeutic abortion for instances of genetic disease if it occurs by a certain point of gestation. In that sense, we are already modifying the population by altering the nature of the genes that are allowed to be carried through to future generations. Dr. Schwob stated, “I wouldn’t want to forbid it categorically because there is a large potential for good.”
The use of CRISPR would require abundant regulation. When asked whether he believes the government should play a role or whether scientists regulate it themselves, Dr. Schwob stated that he believes that the government needs to play a role. The scientists are important in bringing the technology into fruition, but not necessarily the ones concerned with ethical considerations attached to its use. Scientists need to have a voice and make the case for the technology before any regulation is put in place.
In terms of the best use of this technology, Dr. Schwob believes that in the present time, we have already demonstrated examples of success in which there are cells that are taken from the body, modified and put back. This is not a germline alteration but rather a way to cure a disease where there’s a genetic failure to make a proper enzyme or something of that nature. This is no different than the therapy people are pursuing; It is a difference in technology, not the outcome. In terms of the future use, there is a question about the germline modification because it can have far-reaching and long-lasting effects. We must think critically about its nature and whether the law of unintended consequences will intercede and have that be a force that is not good.
The use of CRISPR may lead to unintended consequences so when asked about how to reduce these unintended effects, Dr. Schwob suggested that scientists model the kinds of changes that may occur in animals to see if there are consequences. Understanding more and more about the biology of an organism is the safest way to proceed. There is also the concern that people are desperate for something that can help them and are pushing for something to become available. It is important to be thorough and thoughtful when thinking about treatments.
The use of CRISPR/Cas9 is not limited to germline editing. Dr. Schwob mentioned that some of his colleagues at Tufts University were using CRISPR to cure retinitis pigmentosa, a condition that is caused by a mutation in the rhodopsin gene. An advantage of doing this experiment in the eye is that we already are experienced in injecting drugs into the retina and thus have a lot of understanding on how to physically deliver this system. Good targets for this technology include any diseases that are manifest because of abnormalities of blood cells or cells that derive from the bone marrow.
Dr. Schwob’s lab studies how nervous systems develops and regenerate. They want to see how individual types of progenitor cells contribute to the process of brain developments. His lab used a transgenic mouse to replace an existing sequence so that they could add the gene for fluorescent proteins as a tag to follow specific cell types. In terms of the results, 1 out of 35-40 really had the right mutation, meaning that there is a problem with the fidelity or efficiency of accomplishing genetic change you want. It would be a very expensive and cumbersome technique to apply for human disease at the present time.
Dr. Schwob also presented some of his concerns with this technology. These included, unintended consequences such as by employing this technology are you modifying other parts of the genome? Additionally, we currently don’t know if just the process of having the guide RNA and the protein alters the cell in any way other than the genetic change we want to accomplish.
In terms of the criteria needed before moving forward, Dr. Schwob believes that scientists must demonstrate that this system does not cause any form of harm but that there must be a cost-benefit analysis done before moving forward. There must also be an FDA evaluation process and it must go through different phases of the clinical trial system to demonstrate efficacy that is substantially better than what is available already.
Dr. Schwob also believes that public engagement is very important. He feels very strongly that we need to have a better informed citizenry when it comes to science and medicine. Being ignorant of how these things benefit us will lead to misunderstandings; for example, the kinds of concerns that are not based on reality of the situation but are based on misapprehension.