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Gene Therapy

Gene Editing Success In Primate Embryos

Posted on 2 May 2017

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A US team has effectively and efficiently used CRISPR/Cas9 to edit rhesus monkey embryos, in a proof of concept study

While human embryos have already undergone experimental editing in China, the US is yet to follow suit. Now, a research team has tested the editing system CRISPR-Cas9 on rhesus monkey embryos to test whether primate models could be used as improved human models for the development of new therapies such as gene or cell based therapeutics. 

“Using nonhuman primate embryos is important because the closer we can approximate the human condition in the animal model, the better the chances of developing successful treatments as well as limiting risks that may be encountered in clinical trials. If scientists want to test drugs for dementia, Alzheimer’s or autism, ideal models would react similarly to humans in regards to the reduction of symptoms, outbreak of side effects, such as enduring the same lesions as humans do, or exhibiting similar behavioral characteristics”

While mice remain an excellent and relatively more affordable model for research, primates are much closer to humans and usually provide a more accurate picture of human responses to interventions. Humans often respond very differently to drugs than mice do for example, and as we begin to delve deeper in gene based medicine tinkering with genes in primate embryos provides us with a good indication of how humans might be affected by various treatments. This study currently applies more to the research end of things, as editing adult humans is much more difficult. However, the more proof of principle we have as we move to better models, such as gene edited rhesus monkeys, the closer we move to clinical application of gene editing strategies. Given that primates typically cost around $15,000 each to raise and care for in a period of 4-5 years as they develop into adulthood, the leap in efficiency demonstrated in this study is very encouraging and will likely enable more studies to take place. 

“Extreme amounts of care go into maintaining the well-being of the monkeys. They follow strict protocols to ensure this is a priority. So being able to conduct the science here at Michigan State while partnering with the center is the best combination of science and animal welfare”

Read more at MedicalXpress

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