JUL 17, 2020 7:28 AM PDT

RNA Gene Editing Restores Rett's Syndrome Protein in Mouse Model

WRITTEN BY: Amanda Mikyska

Video:  Overview of how scientists edit an RNA sequence. 

 

Rett's syndrome is a chronic, neurological disorder that causes repetitive hand movements, and a combination of other complications relating to the respiratory system, loss of speech, and musculoskeletal system.  Currently, there is no cure for the disorder, caused by a small error in the DNA code of the MECP2 gene that interferes with the gene producing a functional protein. A new study from the Vollum Institute at the Oregon Health and Science University shows that fixing the RNA code in three different types of neurons restores half the normal amount of the MECP2 protein.

Previous studies from the same researchers show that a synthetic RNA guide can edit mice's genetic code. This study broadens that finding by showing that editing the genetic code is possible in multiple types of neurons and that it achieves the intended downstream effect by restoring MECP2 protein levels. 

Most of Rett's syndrome cases are caused by an A replacing a G in the DNA code, but many possible mutations cause MECP2 to lose its function. Researchers design the guide RNA to match each mouse's specific mutation and administer the guide RNA via an enzyme. The RNA approach was successful in three different types of neurons and restored the function of the gene and the presence of the protein. 

The next step is to inject the guide RNA, via viral vehicle, into the blood so that all cells in the brain would be subject to edit, and to discover side effects.

However, research also showed that the RNA approach resulted in the guide RNA editing places other than the MECP2 gene. No side effects were attributed to the off-target effects, but decreasing off-target edits and increasing editing efficiency is a top priority in future studies.

 

Sources: GEN, NIH, Rett Syndrome Research Trust, Sinnamon et. al.

About the Author
  • Amanda graduated from the University of Massachusetts Boston with a degree in Biology. After working in research on creating biochemicals from genetically engineered yeast, she started freelance science writing while traveling the world. Now, Amanda is a Lab Manager and Research Assistant at the the University of Central Florida, studying the molecular phylogeny of parasitic wasps. She writes about the latest research in Neuroscience, Genetics & Genomics, and Immunology. Interested in working on solutions for food/water security, sustainable fuel, and sustainable farming. Amanda is an avid skier, podcast listener, and has run two triathlons.
You May Also Like
JUL 25, 2021
Genetics & Genomics
Understanding How Mitochondrial Dysfunction May Cause Parkinson's
JUL 25, 2021
Understanding How Mitochondrial Dysfunction May Cause Parkinson's
Parkinson's disease is a complex neurodegenerative disorder. While age is a major risk factor, genetics and environmenta ...
AUG 17, 2021
Neuroscience
When Do I Know I'm Me? Researchers Pinpoint Consciousness Patterns
AUG 17, 2021
When Do I Know I'm Me? Researchers Pinpoint Consciousness Patterns
Anti-correlated neurobiological shifts exist between two brain networks involved in consciousness, the "default mode net ...
AUG 19, 2021
Drug Discovery & Development
Can drugs increase sociability after pediatric brain injury?
AUG 19, 2021
Can drugs increase sociability after pediatric brain injury?
     Traumatic brain injuries (TBIs) are a leading cause of death and disability in children. In the Unit ...
AUG 24, 2021
Health & Medicine
Consciousness is so dop(amine)! Researchers show dopamine plays a key role in consciousness
AUG 24, 2021
Consciousness is so dop(amine)! Researchers show dopamine plays a key role in consciousness
Consciousness is complex, but researchers have found a key element: dopamine
SEP 01, 2021
Health & Medicine
Does Regular Cannabis Affect Brain Function? Sometimes in a Good Way
SEP 01, 2021
Does Regular Cannabis Affect Brain Function? Sometimes in a Good Way
With cannabis now legal in many parts of the world, an increasing number of people now count as regular users of the dru ...
SEP 15, 2021
Neuroscience
Motor Cortex Involved in Vocabulary Learning
SEP 15, 2021
Motor Cortex Involved in Vocabulary Learning
The motor cortex, the part of the brain involved in the planning, control, and execution of voluntary movements, may pla ...
Loading Comments...