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Question

Can already living humans be generically altered/modified in any way?(talking about a fully grown person, not a baby) How far are we from doing that? What have we achieved so far?

Submitted by: Ded Justded


Answer

Expert response from Robert Murray, MD

Professor, Human Nutrition, The Ohio State University

Thursday, 27/12/2018 19:41

Direct replacement of a defective gene with a healthy gene is now being used for disease treatment. The first gene therapy treatment approved by the FDA was for a rare inherited condition that causes progressive blindness starting in childhood. Patients from 4 to 44 yeaers of age were treated by injecting normally functioning genes into the eye. More than 90 percent of those participating showed improvement in vision. In another study, scientists recently reported that they had successfully replaced a gene (CEP290) that causes kidney disease in patients suffering with Joubert’s syndrome. Joubert’s syndrome is a brain disorder that causes physical, mental and sometimes visual impairments. Without treatment, one third of patients will develop kidney failure, forcing on to long-term dialysis or transplantation. The drug should be available within a few years. Even pre-natal gene therapy research was encouraged recently when the National Academy of Sciences indicated that altering the genes of embryos might be allowed under strict criteria if the objective is to prevent serious disease.

 

But this is important to understand: these many new therapies arising from gene-editing technology -- whether it is direct gene-replacement in humans or drugs and vaccines produced using the gene modification tools -- all of these therapies must still undergo the same strict study controls and meet existing regulatory standards to ensure that the treatments are both effective and safe. That will not change in the future.

 

Applying this powerful gene modification technique needs to be performed transparently under stringent guidelines to prevent misuse and unanticipated consequences. Just this week a Chinese scientist claimed that he had used CRISPR to alter embryos before implanting them in a woman, who then gave birth to twin girls. The action appeared to be a violation of China’s scientific regulations and drew immediate condemnation from fellow-scientists, ethicists, the lay public, and Chinese legal representatives. Monitoring the uses of gene editing technology will require a well-informed public. The media and the blogging community will need to step away from the sensationalism that often characterizes public discussion about genetic manipulation and instead provide accurate, scientifically-based perspectives so that the public understands the risks, the benefits, and the process being used.  A good way to keep up with the whole field of genetic modification for biotechnology, agriculture, nutrition, and health is to follow the Genetic Literacy Project at https://GeneticLiteracyProject.org.

 

Answer

Expert response from Community Manager

Moderator for GMOAnswers.com

Thursday, 27/12/2018 20:09

In addition to the response by Robert Murray, Professor, Human Nutrition, The Ohio State University, we have answered similar questions on genetic modification in humans in the past. 

 

Dr. Larry Gilbertson, Ph.D, Genomics Strategy Lead, Monsanto, answered a similar question; an excerpt is included below.

 

Even if germline genetic modification becomes a reality in humans, it is one thing to use it to repair a mutation that causes a disease, but it is quite another to use it to “enhance” human traits.

 

The latter captures people’s imagination, but to do so would require a level of knowledge about complex genetic traits that just doesn’t exist today, so even if the laboratory techniques exist, the data science required to do so doesn’t.

 

Chris Barbey, Ph.D Student, Plant Molecular Genetics and Cell Biology, also answers a similar question; an excerpt from that answer is included below.

 

Ethical considerations aside, gene editing has a large technical hurdle in terms of “delivery.” A human body is made of tens of trillions of cells, and each cell contains the complete genome (some notable exceptions). Currently there’s no method on the horizon for editing all the cells of a large multicellular organism such as ourselves. So even though we know a lot of negative genetics causing human disease, unfortunately we can’t easily correct them in a living person. The technical hurdles preventing the curing of disease are the same hurdles preventing your transformation into Aquaman.

 

We hope this answers your question, if you have any other questions about GMOs or biotechnology, please ask!