D. Joy Riley, M.D., M.A.
Executive Director
This is part IV of our report. View part I, part II, or part III.
The ability to edit genes using CRISPR (clustered regularly interspaced short palindromic repeats) has been in the news for more than a year. A committee has been appointed to advise our government regarding the editing of genes, particularly editing the genes of the human embryo.
Who are the members of that committee? What are their views? The Tennessee Center for Bioethics & Culture has been working to gather information for you, our readers.
Here is a brief look at some of the writings and organizational involvement of the committee members. We continue this month with the final set of seven members:
Keith R. Yamamoto, Ph.D.
University of California, San Francisco
- Bio here
- Projects here
- Human Gene Editing: “A prudent path forward for genomic engineering and germline gene modification,” Science, 2015 (subscription required); co-authors included inter alia Jonathan Weissman and R. Alta Charo
- Embryonic Stem Cell Research: “A New Era in the Ethics of Human Embryonic Stem Cell Research,” Stem Cells, citing need for:
- new human ESC lines
- protection of participants in phase I clinical trials
- respect of donors
Jonathan Weissman, Ph.D.
University of California, San Francisco
- Bio here
- Projects here
- Human Gene Editing:
“The genome is like a piano, says Jonathan Weissman, a biochemist at the University of California, San Francisco. ‘You can play a huge variety of different music with only 88 keys by how hard you hit the keys, what keys you mix up and the timing.’ By dialing down or turning up the activity of combinations of genes at precise times during development, cells are coaxed into becoming hundreds of different types of body cells.
“For the last 20 years, researchers have been learning more about that process by watching when certain genes turn on and off in different cells. Gene activity is controlled by a dizzying variety of proteins known as transcription factors. When and where a transcription factor acts is at least partly determined by chemical tags on DNA and the histone proteins that package it. Those tags are known collectively as epigenetic marks. They work something like the musical score for an orchestra, telling the transcription factor “musicians” which notes to hit and how loudly or softly to play. So far, scientists have only been able to listen to the music. With dead Cas9, researchers can create molecules that will change epigenetic notes at any place in the score, Weissman says, allowing researchers to arrange their own music.” (Science News)
Sharon Terry, M.A.
Genetic Alliance CEO (patient group)
- Human Gene Editing: Ms. Terry was the only patient advocate invited to speak at the December 2015 meeting of the Committee; report here
Ismail Serageldin, Ph.D., M.A.
Bibliotheca Alexandrina
- Bio here
- Publications (list) here and (editorial) here
- Human Gene Editing: “We have been playing god ever since we domesticated plants and animals.” (Washington Post)
Dietram A. Scheufele, Ph.D.
University of Wisconsin-Madison
- Bio here
- Human Gene Editing:
Describing potential dangers here:
“Identifying potential problems or concerns is part of the committee’s charge, and our report will work very carefully through both the scientific complexities of the technology as well as ethical, regulatory or political challenges that might emerge. Many of these challenges are focused on specific applications, such as germline editing. Once germline alterations are introduced into the human population, some have argued, they might be difficult to reverse and to contain within a single community or even country.
“In many ways, the benefits are much more clear-cut, especially when it comes to helping parents whose genome puts their biological children at risk of inheriting certain diseases. Many patient advocacy groups are especially excited about the potential for medical breakthroughs in this arena.”
Janet Rossant, Ph.D.
University of Toronto
- Bio here
- Human Gene Editing: “Even basic research on editing the human germline genome will require very early human embryos, said one expert on IVF. How early? So early that the ‘spare’ embryos in fertility clinics probably won’t work. ‘It will probably be necessary to create embryos’ to develop germline genome editing, said developmental biologist Janet Rossant of the University of Toronto.” (STAT news)
Matthew Porteus, M.D., Ph.D.
Stanford School of Medicine
- Bio here
- Human Gene Editing:
“Matthew Porteus, M.D., Ph.D., associate professor of pediatrics at Stanford University School of Medicine, said that one challenge he has is that as a physician he was taught that the most important person is the patient in front of him. This is the view he brought to the panel’s discussion. On the other hand, the impact of new gene editing technology reaches far beyond a single patient, he noted. ‘If we could give parents a safe and effective way of making sure that their kids were not going to have diseases, then as an MD, that is absolutely something that we should be able to do.'” (MedPageToday.com article)
“It is important to distinguish between editing that results in germ-line transmission and editing of somatic cells or stem cells (such as SSCs or embryonic stem cells) that can create germ cells. There is no controversy over the potential of curing patients by editing of somatic cells, and it is critical that the scientific community speak with a clear voice on this application. There is less consensus on the experimental use of editing to probe the biology of human germ cells. Nonetheless, we believe that it would be a serious setback to the biomedical research endeavor to preclude the experimental use of this transformative strategy to study fundamental germ cell biology. It is also important for the scientific community to clearly distinguish between discovery-based editing of germ cells and potential applications that would create humans.” (Quote from Nature)
Q&A: Readers’ Questions on CRISPR
What is new with CRISPR?
- Recent overview here
- Sickle Cell Disease and CRISPR
- One of the more recent reports comes from China: a safety trial using CRISPR treatment for metastatic non-small-cell lung cancer has begun, and 10 patients are expected to be enrolled. Other cancer trials are planned in the US and in China; see the report here.
Where does Francis Collins, M.D., Ph.D., Director of The National Institutes of Health, stand regarding germline gene editing?
In August 2016, he enumerated several concerns about germline gene editing (quote from MedPageToday.com):
- Off-target effects of the technology could cause unanticipated mutations;
- Lack of consent from future generations whose genome will be altered;
- Lack of consensus regarding the meaning of enhancement (i.e., the threat of eugenics);
- Fear of the “commodification” of children;
- Rejection of disability as a suitable outcome; and
- Unequal access due to socioeconomic factors.And for those who believe in God, Collins said, the technology raises philosophical and theological questions, around “humans taking charge of our own instruction books” — “I am totally bullish about somatic cell gene editing for clinical benefit, and I think we should be pushing that at maximum speed and energy,” he said.
To submit a question of your own, contact us.
Update
As of 14 February 2017, the National Academy of Sciences and the National Academy of Medicine released a report that indicates “clinical trials for genome editing of the human germline – adding, removing, or replacing DNA base pairs in gametes or early embryos – could be permitted in the future, but only for serious conditions under stringent oversight.” See the press release here.