CRISPR-Mediated Gene Editing as a Novel Approach for Parkinson’ s Disease Therapy
By Neuroscience club Edited by Arina Evtushenko
An estimated 10 million people worldwide currently live with Parkinson’ s disease( PD), a neurodegenerative disorder. Many treatments and medications reduce its symptoms, but no cure has been found. CRISPR— a relatively new gene-editing tool— has proved valuable for researching the genetic mutations behind PD, and several labs have already used it in ways that hint it could one day treat, and perhaps cure, the disease.
What is CRISPR?
CRISPR stands for“ clustered regularly interspaced short palindromic repeats.” It is a technology scientists use to selectively modify the DNA of living organisms, adapted from genome-editing systems found naturally in microbes. These microbes use CRISPR as part of their immune system: when a virus enters a bacterial cell, the bacterium incorporates some of the virus’ s DNA into its own genome so it can recognise and eliminate that virus during future infections— much like antibodies in humans.
CRISPR – Cas9 is an enzyme that can cut DNA. It carries a guide RNA( gRNA) that helps it find the target gene; the enzyme then unzips the DNA, the guide RNA binds to the complementary sequence through base pairing, and Cas9 cuts the target. From there, CRISPR can do one of three things: introduce a new mutation( for example, cutting out a disease-causing sequence and replacing it with a harmless one), delete an unwanted gene by removing the sequence and rejoining the DNA ends, or insert an entirely new section of DNA. Currently CRISPR is mostly a research tool, but scientists are developing CRISPR-mediated gene therapies for several genetic diseases— offering hope for more efficient, more effective treatments of conditions that are presently incurable.
Parkinson’ s disease
Parkinson’ s is a progressive brain disorder that damages dopamine-producing neurons, causing them to weaken, become damaged and die.
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