New gene editing tools let scientists alter the DNA of living cells - from plants, animals, even humans - more precisely than ever before. Safety is a key question because gene editing isn't always precise enough; there's the possibility of accidentally cutting DNA that's similar to the real target.
In this photo provided by Oregon Health & Science University, Shoukhrat Mitalipov, left, talks with research assistant Hayley Darby in the Mitalipov Lab at OHSU in Portland, Ore. Mitalipov led a research team that, for the first time, used gene editing to repair a disease-causing mutation in human embryos.
New gene editing tools let scientists alter the DNA of living cells – from plants, animals, even humans – more precisely than ever before. Think of it as a biological cut-and-paste program. A look at the science.
WHAT IS GENE EDITING
While scientists have long been able to find defective genes, fixing them has been so cumbersome that it’s slowed development of genetic therapies. There are several gene editing methods, but a tool called CRISPR-Cas9 has sparked a boom in research as laboratories worldwide adopted it over the past five years because it’s faster, cheaper, simple to use with minimal training and allows manipulation of multiple genes at the same time.
HOW IT WORKS
Pieces of RNA are engineered to be a guide that homes in on the targeted stretch of genetic material. The Cas9 is an enzyme that acts like molecular scissors to snip that spot. That allows scientists to delete, repair, or replace a particular gene.
MEDICAL RESEARCH
The fresh attention comes from research involving human embryos. In laboratory experiments, a team lead by Oregon researchers used CRISPR to successfully repair a heart-damaging gene in human embryos, marking a step toward one day being able to prevent inherited diseases from being passed on to the next generation. But there’s wide agreement that more research is needed before ever testing the technique in pregnancy.
The biggest everyday use of CRISPR so far is to engineer animals with human-like disorders for basic research, such as learning how genes cause disease or influence development and what therapies might help.
But promising research, in labs and animals so far, also suggests gene editing might lead to treatments for such diseases as sickle cell, cancer, maybe Huntington’s _ by altering cells and returning them to the body. Another project aims to one day grow transplantable human organs inside pigs.
In this microscope photo provided by Oregon Health & Science University, human embryos grow in a laboratory for a few days after researchers used gene editing technology to successfully repair a heart disease-causing genetic mutation. (Oregon Health & Science University via AP)
THE BIGGEST HURDLE
Safety is a key question because gene editing isn’t always precise enough; there’s the possibility of accidentally cutting DNA that’s similar to the real target. Researchers have improved precision in recent years, but out-of-body treatments like using cells as drugs get around the fear of fixing one problem only to spark another.
THE ETHICS CONTROVERSY
Altering genes in sperm, eggs or embryos can spread those changes to future generations, so-called “germ line” engineering. But it’s ethically charged because future generations couldn’t consent, any long-term negative effects might not b
In this photo provided by Oregon Health & Science University, Shoukhrat Mitalipov, left, talks with research assistant Hayley Darby in the Mitalipov Lab at OHSU in Portland, Ore. Mitalipov led a research team that, for the first time, used gene editing to repair a disease-causing mutation in human embryos.
WHAT IS GENE EDITING
HOW IT WORKS
Pieces of RNA are engineered to be a guide that homes in on the targeted stretch of genetic material. The Cas9 is an enzyme that acts like molecular scissors to snip that spot. That allows scientists to delete, repair, or replace a particular gene.
MEDICAL RESEARCH
The fresh attention comes from research involving human embryos. In laboratory experiments, a team lead by Oregon researchers used CRISPR to successfully repair a heart-damaging gene in human embryos, marking a step toward one day being able to prevent inherited diseases from being passed on to the next generation. But there’s wide agreement that more research is needed before ever testing the technique in pregnancy.
The biggest everyday use of CRISPR so far is to engineer animals with human-like disorders for basic research, such as learning how genes cause disease or influence development and what therapies might help.
But promising research, in labs and animals so far, also suggests gene editing might lead to treatments for such diseases as sickle cell, cancer, maybe Huntington’s _ by altering cells and returning them to the body. Another project aims to one day grow transplantable human organs inside pigs.
In this microscope photo provided by Oregon Health & Science University, human embryos grow in a laboratory for a few days after researchers used gene editing technology to successfully repair a heart disease-causing genetic mutation. (Oregon Health & Science University via AP)
THE BIGGEST HURDLE
Safety is a key question because gene editing isn’t always precise enough; there’s the possibility of accidentally cutting DNA that’s similar to the real target. Researchers have improved precision in recent years, but out-of-body treatments like using cells as drugs get around the fear of fixing one problem only to spark another.
THE ETHICS CONTROVERSY
Altering genes in sperm, eggs or embryos can spread those changes to future generations, so-called “germ line” engineering. But it’s ethically charged because future generations couldn’t consent, any long-term negative effects might not b
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