Innovative Gene Therapies for Obesity: Focusing on MC4R Deficiency

A complex health issues, obesity is impacted by both genetic predispositions and dietary habits.. With respect to the most important genes involved is MC4R gene (Melanocortin 4 Receptor), critical for appetite control and energy balance. Disfunction of this gene, and especially when present during early life, may lead to chronic hunger, lower expenditure of energy, and marked weight gain that becomes difficult to manage.Standard treatment protocols for obesity have involved lifestyle change like exercise and diet, with pharmacological intervention in the form of orlistat (Alli, Xenical) and phentermine-topiramate (Qsymia). Nonetheless, these measure little in response in individuals carrying MC4R mutations. Drugs such as liraglutide (Saxenda) and bupropion-naltrexone (Contrave) have been shown to have little effect on the root problems behind these genetic mutations, emphasizing the need for more specific medicines.Gene therapy that delivers brain-derived neurotrophic factor (BDNF) through adeno-associated virus (AAV) vectors is a promising treatment for MC4R deficiency. .This review highlights the significance of genetic profiling to select obese patients with MC4R mutations, which can improve and personalize treatment regimens. By learning the particular genetic basis of obesity in these patients, medical professionals can make interventions more likely to be effective. In general, although gene therapy offers a new method for addressing obesity due to MC4R deficiencies, additional research is needed to improve delivery systems and reduce the risk of immunological reactions. Further investigation in this area promises to enhance outcomes for those suffering from this multifaceted disorder.

Publication Details

Journal
Zenodo (CERN European Organization for Nuclear Research)
Published
2025-01-01
DOI
https://doi.org/10.5281/zenodo.16981822
Primary Topic
Geochemistry and Geologic Mapping
Type
article
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article

Innovative Gene Therapies for Obesity: Focusing on MC4R Deficiency

Zenodo (CERN European Organization for Nuclear Research)
Geochemistry and Geologic Mapping
article

Innovative Gene Therapies for Obesity: Focusing on MC4R Deficiency

article en

Abstract

A complex health issues, obesity is impacted by both genetic predispositions and dietary habits.. With respect to the most important genes involved is MC4R gene (Melanocortin 4 Receptor), critical for appetite control and energy balance. Disfunction of this gene, and especially when present during early life, may lead to chronic hunger, lower expenditure of energy, and marked weight gain that becomes difficult to manage.Standard treatment protocols for obesity have involved lifestyle change like exercise and diet, with pharmacological intervention in the form of orlistat (Alli, Xenical) and phentermine-topiramate (Qsymia). Nonetheless, these measure little in response in individuals carrying MC4R mutations. Drugs such as liraglutide (Saxenda) and bupropion-naltrexone (Contrave) have been shown to have little effect on the root problems behind these genetic mutations, emphasizing the need for more specific medicines.Gene therapy that delivers brain-derived neurotrophic factor (BDNF) through adeno-associated virus (AAV) vectors is a promising treatment for MC4R deficiency. .This review highlights the significance of genetic profiling to select obese patients with MC4R mutations, which can improve and personalize treatment regimens. By learning the particular genetic basis of obesity in these patients, medical professionals can make interventions more likely to be effective. In general, although gene therapy offers a new method for addressing obesity due to MC4R deficiencies, additional research is needed to improve delivery systems and reduce the risk of immunological reactions. Further investigation in this area promises to enhance outcomes for those suffering from this multifaceted disorder.

Zenodo (CERN European Organization for Nuclear Research)
Zero hunger
Openalex Percentile: Top 63%
Geochemistry and Geologic Mapping
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Innovative Gene Therapies for Obesity: Focusing on MC4R Deficiency · Zenodo (CERN European Organization for Nuclear Research) (2025) | TGRS Research Map | TGRS