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A Boost of Klotho Protein Level Has Therapeutic Potential in Amyotrophic Lateral Sclerosis (ALS), Also Known as Lou Gehrig's Disease
BOSTON, June 27, 2019 /PRNewswire/ -- In a paper published today, scientists report that increasing the levels of the naturally occurring Klotho protein reduces the neurological deficits and prolongs life span in an experimental model of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. The lead scientists on this study are affiliated with Klogene Therapeutics, Inc. Klogene is developing therapies based on increasing the level of Klotho protein. The study was performed at Boston University School of Medicine. The paper was published in the Journal of Molecular Neuroscience (https://link.springer.com/article/10.1007/s12031-019-01356-2).
ALS is a devastating neurodegenerative disease, characterized by the loss of upper and lower motor neurons, leading to progressive muscle atrophy and paralysis, which is fatal within three to five years of diagnosis.
Klotho is a naturally occurring, anti-aging pleiotropic human protein/hormone that has been shown to have potential therapeutic effects in neurodegenerative diseases, kidney disease, certain cancers and more. Klotho levels typically decrease with age. Preventing Klotho level decline, or increasing it slightly above its level at a young age, is protective, i.e. it increases resilience to several pathologies associated with aging.
"I am excited about the results in this mouse model of the disease and hope that similar benefits will eventually be achieved in ALS patients," said Dr. Ella Zeldich, the lead Boston University scientist on the project and a Klogene Therapeutics, Inc. advisor.
"We have been studying Klotho for 16 years and reported in 2008 that Klotho declines in the brain of non-human primates as they age. We suggested back then that a boost in Klotho may have therapeutic potential in neurodegenerative diseases. Since then, several studies in our lab and by others have supported our hypothesis. Our recent exciting results in ALS are the last in a series of such validating studies," said Dr. Carmela Abraham, professor of biochemistry, pharmacology and experimental therapeutics at Boston University School of Medicine and the head of the lab in which this study was performed. Dr. Abraham is also a Klogene co-founder and advisor.
"Klogene developed several modalities for increasing endogenous Klotho protein level or supplementing it. Our research and intellectual property include small molecules, zinc finger proteins, RNA targeted compounds, activation with CRISPR dCas9 tools and gene therapy," said Dr. Cidi Chen, senior research scientist at Klogene Therapeutics, Inc.
"We are pleased to be able to add ALS to the list of debilitating diseases that Klogene's patent-pending technologies can target. Other indications include cognitive impairment, dementia, Alzheimer's, multiple sclerosis, kidney disease, certain cancers, and more," said Menachem Abraham, co-founder, chairman and CEO of Klogene. "We seek the optimal Klotho boost therapeutic modality for each of these diseases," he added.
About Klogene Therapeutics, Inc.
Klogene is developing therapies based on increasing the level of Klotho protein.
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SOURCE Klogene Therapeutics, Inc.