One and Done Gene Therapy

Does Gene Therapy for Obesity Offer a Possible Cure?

DNA LabNew research is pointing to the possibility that gene therapy might eventually offer a cure for people with some forms of obesity. Publishing their research in Cell Reports, Akinori Takahashi and colleagues have found that by manipulating two genes in mice — Cnot7 and Tob — they could make mice burn more of their fat stores and become resistant to obesity.

Of course, this is a long way from providing a therapy for clinical use in people, but that is where these researchers are headed:

Cnot7 and Tob may function as attenuators of adaptive thermogenesis under a high-calorie regimen, raising the possibility that Tob and Cnot7 could be useful therapeutic targets for obesity, because of their ability to destabilize Ucp1 mRNA.

Right now we are resigned to managing obesity as a chronic disease. Even with bariatric surgery, which sometimes provides dramatic improvements, the realistic expectation is that it only puts this chronic disease into remission. Which means that relapses can happen, as people who have been there know all too well. But progress in gene therapy for other diseases makes its application in obesity seem less like science fiction. Reporting on gene therapy for inherited blood disorders this week, Harvard Professor Marcela Maus said:

I am thrilled with these results. It seems like gene therapy is becoming a clinical reality. There have been a lot of challenges in this field, but the new results are showing both efficacy and safety.

Last year, Mingming Lao and Dexi Liu summarized the promise and challenges of gene therapy for obesity, saying:

Unlike cancer or single-gene deficiency-related genetic diseases, obesity is a chronic disease that is not lethal but may lead to medical complications. Careful assessment of the long term effects of gene transfer is absolutely necessary to ensure the safety of the expression of the therapeutic gene. Despite these challenges, it is predictable that the gene therapy based strategy in modulating metabolism and treating metabolic disorders will surely impact how we live a healthy life.

We look forward to progress on this front.

Click here for the study in Cell Reports, here for more from Medical Daily, and here for the review by Lao and Liu.

One and Done Gene Therapy, photograph by Luis Tecedor and Beverly L. Davidson at Children’s Hospital of Philadelphia, courtesy of NIH Image Gallery on flickr

Subscribe by email to follow the accumulating evidence and observations that shape our view of health, obesity, and policy.


December 19, 2015