Six Conicet science teams and universities use molecular scissors to develop therapies for human diseases, pet fractures and to replace transgenic crops. Infobae spoke with specialists in depth in their innovative methods
Six scientific groups in Argentina are using the most modern genetic editing technique to give answers to patients with potentially deadly diseases, pets and agricultural producers. The technique – known by its initials as CRISPR – allows cutting and pasting DNA without adding genes from other species. And they already manage it in Conicet labs and public universities to offer more effective treatments for diabetes, deafness, cow’s milk allergy, heart disease (which can lead to sudden death), hereditary Alzheimer’s disease, and Melanoma, the most aggressive skin cancer. They expect to have human-compatible pigs that need transplants, and drugs that will come in the vaccine milk. At the moment, none work with human embryos.
The CRISPR technique is within the range of genetic therapies that had been promised by science in the 1990s. At that time, the technology available was ineffective and unsafe for patients. That is why, now the hope is in CRISPR, which somehow mimics the system of defenses of bacteria to combat the virus attack. In Argentina, the six scientific groups look for ways to adapt the technique according to different objectives.
The scientist Adrián Mutto and his collaborators at INTA de Balcarce, Nicolás Mucci and Germán Kaiser, are anxiously awaiting the arrival of February 2018, at which time the birth of the children of seven Holando cows will occur. These animals could produce milk without allergens. “Some people are allergic to the beta-lactoglobulin protein, and they can not consume dairy. For them, we are using genetic engineering, which will enable cows to not have protein,” said Mutto. “They will not be transgenic,” said the scientist who works at the Biotechnology Research Institute, which depends on Conicet and the National University of San Martin. In addition, they are in the process of requesting authorization from the National Advisory Committee on Agricultural Biotechnology to generate modified pigs as models for the study of human diseases, such as cancer, diabetes, heart disease, and pulmonary diseases. Another goal is to generate other modified pigs with a superior yield in the quantity of meat, with a view to the consumer market.
With his research group from Conicet at the University Hospital of the Italian Hospital in Buenos Aires, Federico Pereyra Bonnet used the technique of genetic editing to turn off or turn on the genes of a human skin cell and turn it into an insulin-producing cell. He has in mind the development of a treatment for people with diabetes. Now, they are evaluating the efficacy and safety of potential CRISPR therapy in mice that are suffering from this disease in an induced manner. “There is still a long way to go for patient therapy,” Pereyra Bonnet told Infobae. In addition, they have two other projects in progress: developing hearing cells for deaf people and bone-building cells to quickly weld fractures of pets like dogs.
“The technique is so simple that we could develop it here without the need to receive training abroad. The tool we used before cost us $ 8,000, while CRISPR only invested 150,” said Pereyra Bonnet. They published works in Gene Therapy and in Stem Cell International, and continues with funds from the Italian Hospital and the National Agency for Scientific and Technological Promotion. That organism is also supporting the combined use of genetic editing with reprogrammed stem cells in the Fleni, where researcher and cardiologist Santiago Miriuka attempts to reproduce in the laboratory how a rare heart problem occurs, which is known as “arrhythmogenic dysplasia Right ventricle, “and the hereditary form of Alzheimer’s disease. “We are creating disease models in the laboratory,” Miriuka told Infobae.
One of the “parents” of the first calf clone that was born in Argentina in 2002, scientist Daniel Salamone, had succeeded in eliminating by CRISPR the mad cow gene in embryos last year and now continues in other areas. “We have public subsidies to produce modified pigs that can be used to produce organs for patients who need transplants, to develop drugs in cows’ milk and to improve cloning techniques,” said Infobae Salamone, a new member of the National Academy of Veterinary.
In Rosario, there are two groups that work with possible benefits for patients and agricultural producers. Nora Calcaterra, PhD in biochemistry and researcher at the Institute of Molecular and Cellular Biology of Rosario, who depends on Conicet and the National University of Rosario, is editing the embryo genome of the zebrafish. It does so with the idea of finding the molecular mechanisms that lead to the differentiation of skin cells, which are called melanocytes. “We hope to better understand the mechanisms that lead to the development of more aggressive skin cancer, melanoma. Preliminary results are encouraging,” Calcaterra said.
In the same institute in Rosario, Javier Palatnik also insists on taking advantage of the genetic edition. He does it with plants. It tests with the most used vegetable species in the laboratories that is Arabidopsis thaliana, and tries to make modifications in the genome to get to transfer the knowledge to the crops of commercial interest, like soybean or wheat. “We are looking to increase crop productivity and make it more resistant to the stress of drought. If the results are positive, there is a possibility that GM crops will stop being produced to be replaced by mass production with new genetic engineering techniques” . The end of transgenics could be close, but we still need to be sure about the organisms that will replace them.