Research on gene therapy continues to uncover remarkable benefits. Its greatest promise is its unrivaled potential to treat many diseases currently considered incurable.

Amyotrophic lateral sclerosis (ALS) is a disease marked by a progressive loss of motor neurons. The prognosis for this disease is paralysis and death. Until recently, no treatments have been deemed hopeful for the fight against ALS. However, this has changed, as a 2019 study on the effects of Klotho on an ALS mouse model yielded remarkable results.

Klotho overexpression in the ALS mouse model delayed both the onset and progression of the disease, resulting in an extended lifespan. Klotho prevented neuronal loss and reduced the expression of neuroinflammatory markers. Furthermore, it enhanced promyelinating and antioxidative factors in both the motor cortex and spinal cord. (Zeldich, 2019)

Discoveries like this may come as a surprise to those who are unfamiliar with the astounding results from a multitude of studies on klotho. Multiple studies have demonstrated that klotho overexpression enhances cognition and extends life span both in animal models and in humans. (Abraham, 2016)

Indeed, Klotho may have phenomenal benefits for our brain. Higher Klotho levels are linked to higher IQs in older people, as well as less cognitive decline. (Shimokata, 2006) Furthermore, as Klotho regulates functions ranging from cognition, neurogenesis, and synaptic plasticity, it is rightly viewed as a potential therapy to protect against neurodegenerative diseases including Alzheimer’s. (Vo, 2018)

Klotho stands as a lodestar in modern medicine. As a potential game-changer for rare diseases like ALS and more common maladies like Alzheimer’s, it is giving hope to millions of people. As a general means to attain a longer and healthier life while keeping a sharp mind, it may be a gift to us all.

References and Suggested Reading:

Zeldich E, Chen CD, Boden E, Howat B, Nasse JS, Zeldich D, Lambert AG, Yuste A, Cherry JD, Mathias RM, Ma Q, Lau NC, McKee AC, Hatzipetros T, Abraham CR. Klotho Is Neuroprotective in the Superoxide Dismutase (SOD1G93A) Mouse Model of ALS. J Mol Neurosci. 2019 Oct;69(2):264-285. doi: 10.1007/s12031-019-01356-2. Epub 2019 Jun 27. PMID: 31250273; PMCID: PMC7008951

Abraham CR, Mullen PC, Tucker-Zhou T, Chen CD, Zeldich E. Klotho Is a Neuroprotective and Cognition-Enhancing Protein. Vitam Horm. 2016;101:215-38. doi: 10.1016/bs.vh.2016.02.004. Epub 2016 Mar 22. PMID: 27125744.

Shimokata H, Ando F, Fukukawa Y, Nishita Y. Klotho gene promoter polymorphism and cognitive impairment. Geriatr Gerontol Int. 2006;6(2):136–41

Vo, Hai T et al. “Klotho, the Key to Healthy Brain Aging?.” Brain plasticity (Amsterdam, Netherlands) vol. 3,2 183-194. 10 Aug. 2018, doi:10.3233/BPL-170057

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