RNA editing promises to go where DNA editing can’t
On October 16, a biotechnology company in Massachusetts in the U.S. named Wave Life Sciences made headlines for becoming the first company to treat a genetic condition by editing RNA at the clinical level. RNA editing allows scientists to fix mistakes in the mRNA after the cell has synthesised it but before the cell reads it to make the proteins. RNA editing in development Wave Life Sciences used RNA editing to treat α-1 antitrypsin deficiency, an inherited disorder. Wave Life Sciences is planning to extend its RNA editing technology to treat Huntington’s disease, Duchenne muscular dystrophy, and obesity. Some other companies using ADAR enzymes to perform RNA editing are Korro Bio for AATD and Parkinson’s disease; ProQr Therapeutics for heart disease and bile acid build-up in the liver; and Shape Therapeutics for neurological conditions.
Discover Related
The controversial scientist He Jiankui hasn’t given up on gene-edited babies
Can gene editing help with hereditary eye diseases? A new study explores the possibility
S. Africa’s new research guidelines not a green light for heritable human genome editing
Editing fetal genomes is on the horizon. We need to talk about it now
Ethical guideline issued for genome editing
Guideline sets ethical rules for human genome editing research
Gene therapy trials treat inherited blindness and deafness
With CRISPR poised to revolutionise therapy, a pause to consider ethical issues
For man behind gene-edited babies, a rocky return to science
Documentary spurs a new look at the case of the first gene-edited babies
Could science finally be able to treat genetic disorders?
Humans could be 'gene edited' to cure chronic conditions
Gene-Editing Experiment Improves Sight In Patients With Impaired Vision
New CRISPR tech can revolutionise gene therapy, renew hope for treating genetic diseases
Gene editing techniques helpful in retinal degeneration treatment
Gene-editing treatment shows promise for sickle cell disease
CRISPR gene editing can cause unwanted changes in human embryos, study finds
Still too soon to try altering human embryo DNA, panel says
It’s still too soon to try altering the DNA of human embryos, scientific panel says
Never-seen-before extracts from 'outrageous' research of disgraced scientist He Jiankui
CRISPR Gene Editing May Offer Path To Cure For HIV, First Published Report Shows
For the first time, researchers use gene editing to cure mice of HIV
Gene Editing May Treat Lethal Lung Diseases Before Birth: Study
CRISPR used to treat two human cancer patients for the first time in UPenn study
First U.S. Patients Treated With CRISPR As Human Gene-Editing Trials Get Underway
Scientists in China are losing touch with patients of gene editing trials: Report