Scientists from Boston University School of Medicine and Klogene Therapeutics have made precision medicine news this week by showing efficient transcriptional activation of the anti-aging multifunctional gene Klotho by CRISPR-dCas9, as reported in a paper published today in the "Journal of Molecular Neuroscience."
CRISPR is a gene editing tool first introduced six years ago. It works like a pair of scissors that can cut DNA, inserting or reordering bits of genetic code. Currently, it is being used in cancer treatment clinical trials. Scientists expect more clinical trials in the next five years.
[Also:New CRISPR version could make precision medicine even more precise]
Klotho is a naturally occurring pleiotropic human protein that has been shown to have potential therapeutic effects in neurodegenerative diseases such as Alzheimer's as well as multiple sclerosis, kidney disease, certain cancers and others. Higher levels of Klotho are associated with longevity and improved cognitive functions.
"We are excited to be the first to demonstrate feasibility of CRISPR-based Klotho gene regulation using single-guided RNA, targeting the promoter region," said Cidi Chen, MD, senior research scientist at Klogene and a research associate professor at Boston University School of Medicine.
[Also:Regenerative medicine, gene editing markets are growing]
Klogene has assembled a team with skills in small molecule drug development, gene therapy, genome editing and biologics and is applying all of these capabilities, and its technologies, to the company's mission of curing debilitating diseases by enhancing the expression of the Klotho gene, said Menachem Abraham, founder, chairman and CEO of Klogene which has signed a number of research agreements with academic institutions and a small biotech firm.
"We have active collaborations, including pre-clinical studies in disease models, in Europe, Israel and the United States," Abraham said. "In addition to our own lab, on the Boston University School of Medicine campus, we make extensive use of contract research organizations, leading academic laboratories in our field, as well as other focused biotech startups."
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