Advancements in science have enabled researchers to design an ex-vivo gene therapy that can combat obesity and diabetes in mice. This therapy is designed to implant cells that have been manipulated and transformed into treating diseases. The study published in the ‘Metabolic Engineering’ journal is the first to apply ex-vivo gene therapy to generate and implant cells expressing the CPT1AM protein, which plays a crucial role in metabolic diseases such as obesity. The therapy approach will help reduce obesity and glucose intolerance in mice and can be useful in other therapeutic approaches.
The study is led by Professor Laura Herrero and her team from the University of Barcelona and the Center for Network Biomedical Research in the Physiopathology of Obesity and Nutrition (CIBERobn). They utilize stem cells that differentiate and become adipocytes and implanted the cells into animal models to express an active form of the CPT1AM enzyme. The process of cell transformation takes place outside the organism’s body, making this therapy much easier to perform while allowing greater control of the altered cells.
The researchers observed promising results of reduced weight, fatty liver (hepatic steatosis), cholesterol, and glucose levels in obese mice. Adipose tissue plays a significant role in regulating energy balance. Therefore, adipose-derived mesenchymal stem cells have gained interest in cell therapy to help combat these metabolic disorders. The study supports the future clinical use of this ex-vivo gene therapy approach as a new strategy to reduce obesity rates and cholesterol in the population.
To approach therapy in humans, the researchers need to optimize several processes, such as the viability and quality of stem cells from adipose tissue isolated from people with obesity, the percentage of lentivirus infection, and the number of cells used for transplantation.
The above-mentioned study shows that obesity and related metabolic disorders have become a global health and social problem, and new therapeutic approaches are urgently needed. The advent of technological advancements and medical innovations has also enabled other similar beneficial research studies.
Gene therapies have come a long way since their inception, and the prospect of gene editing and the ability to alter a person’s DNA makes them controversial. However, gene therapy has shown success in pre-clinical trials for different diseases.
Scientists in the United States have developed CRISPR gene editing, becoming a powerful tool that can precisely target and alter DNA. CRISPR can target the faulty portion of a person’s genome and cut it out, replacing it with a correct sequence that would, in theory, cure the disease. Medical researchers are now working on developing this technique to combat cancer.
Medical innovations and research studies surrounding the topic have led to improved treatment of patients with different ailments. It can be said with certainty that gene therapies have come a long way since their early developments in the 1970s.
Summary:
Researchers have developed a new ex-vivo gene therapy that combats obesity and diabetes in mice by implanting cells that have been manipulated and transformed to treat the disease. This therapy is less invasive and easier to perform, and researchers noticed promising results of reduced weight, fatty liver, cholesterol, and glucose levels in obese mice. The study supports the future clinical use of this ex-vivo gene therapy approach as a new strategy to reduce obesity rates and cholesterol in the population. Gene editing and altering DNA via CRISPR are advanced medical technologies that can precisely target and alter DNA to cure diseases. Medical innovations and research surrounding these studies have led to improved treatment options for different ailments.
Gene therapies have come a long way since their early development in the 1970s. As technology and medical advancements continue, researchers will better understand these therapies’ capabilities for treating different diseases. Researchers believe there is much potential for using gene therapy as an essential therapeutic approach to combat many different ailments. It is an exciting time in medicine as researchers continue to explore and develop gene therapies that may soon impact the cure for many diseases.
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A scientific team from the University of Barcelona and CIBERobn has designed a strategy to combat obesity and diabetes in mice through ex vivo gene therapy that consists of implanting cells that have been manipulated and transformed to treat a disease. This is the first study applying the ex vivo gene therapy technique to generate and implant cells that express the CPT1AM protein, an enzyme that plays a decisive role in many metabolic diseases such as obesity.
The study, published in the journal metabolic engineeringIt is led by Professor Laura Herrero, from the Faculty of Pharmacy and Food Sciences and the Institute of Biomedicine of the University of Barcelona (IBUB), and the Center for Network Biomedical Research in the Physiopathology of Obesity and Nutrition (CIBERobn). .
Cell therapy describes the process of introducing new cells into a tissue to fight a disease. Cell therapies are currently focused on the treatment of hereditary diseases, with and without the help of gene therapy, or degenerative diseases.
“In this new therapy, stem cells derived from adipose tissue have been implanted subcutaneously in animal models, differentiated into adipocytes, so that they can express an active form of the CPT1AM protein, an enzyme located in mitochondria that is key in oxidation lipid and is related to metabolic diseases”, points out Laura Herrero, member of the Department of Biochemistry and Physiology of the UB.
“As a result, in obese mice it has been possible to reduce weight, fatty liver (hepatic steatosis), cholesterol and glucose levels. In conclusion, implantation of adipocytes expressing the mitochondrial enzyme CPT1AM helps to reduce obesity and glucose intolerance in mice.”
As the cell transformation process takes place outside the body of the organism, this type of therapy is much easier to perform and allows greater control of the altered cells.
Obesity and cell therapy
Obesity and associated metabolic disorders represent a global health and social problem, which is why new therapeutic approaches are urgently needed. Adipose tissue plays a key role in regulating energy balance, and adipose-derived mesenchymal stem cells (cells with self-renewal capacity) have gained interest in cell therapy.
“Specifically, carnitine palmitoyltransferase 1A (CPT1A) is the enzyme that controls the oxidation of mitochondrial fatty acids. Our goal was to generate adipocytes that could express a constitutively active form of CPT1A, CPT1AM, capable of burning excess fat and improving metabolic phenotype of obesity of mice after implantation”.
The results of the new study support the future clinical use of this ex vivo The gene therapy approach as a new strategy to reduce obesity and cholesterol rates in the population.
This preclinical study could open the doors to future therapeutic strategies to address the treatment of obesity, which today represents a global health problem.
“To approach therapy in humans, we need to optimize several processes such as the quality and viability of stem cells from adipose tissue isolated from people with obesity, the percentage of lentivirus infection, and the number of cells used for transplantation,” he concludes. researcher Laura Herrero.
https://www.sciencedaily.com/releases/2023/05/230530125435.htm
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