Study co-author
Stephen Tsang, M.D., Ph.D., associate professor of
ophthalmology, pathology, and cell biology at CUMC, Columbia's
Institute of Genomic Medicine, and the Institute of Human
Nutrition, said:
"We feel
it's critical that the scientific community consider the
potential hazards of all off-target mutations caused by CRISPR,
including single-nucleotide mutations and mutations in
noncoding regions of the genome." [2]
CRISPR alters
specific DNA sequences, but fails to correct the side effects
that occur as a result.
There are
currently 2 clinical trials involving humans underway in China,
and a U.S. trial is slated to begin sometime next year. As many
as 20 trials are in the works.
One of the
trials is aimed at preventing cervical cancers by using CRISPR
to target and destroy the genes of the human papillomavirus (HPV)
that cause tumors. [1] [3]
Here is a video
talking a little bit about CRISPR.
Scientists
identify areas likely to be affected by off-target DNA
mutations with the assistance of predictive computer
algorithms.
Refinements
to the technology suggested that in those trials off-target
effects would be few. But nobody knows for certain how many
side effects will result, or how serious they will be, and
many experts are convinced the technology is simply not safe
for use in humans at this point in time. [2]
Alexander Bassuk, M.D., Ph.D., the study's co-author and
professor of pediatrics at the University of Iowa, said:
"These
predictive algorithms seem to do a good job when CRISPR
is performed in cells or tissues in a dish, but
whole-genome sequencing (WGS) has not been employed to
look for all off-target effects in living animals."
In the
experiment in mice, researchers learned that success in the
lab doesn't always translate into success in the real world.
The team looked at mice that they had previously corrected a
blindness-associated gene in and sequenced their entire
genome to find changes.
Though the
mice's vision was restored, 2 of them were left with over
1,500 unintended mutations, and more than 100 deletions and
insertions of genes the researchers never intended to touch.
What's
more, the computer algorithm failed to predict any of
them. [1]
Outwardly,
the mice didn't look any different or develop any
superpowers. But how the mutations will impact the mice in
the future, or in subtle ways, remains an unanswered
question for now.
The vast
majority of genetic mutations are not good. They're exactly
what CRISRP is intended to treat.
One scientist warned in January 2017 that
gene-editing has the potential to "ruin human evolution."
Obviously, others see it as a promising advancement for the
future.
The
researchers concluded:
"This
finding warns that CRISPR technology must be further
tailored, particularly before it is used for human
gene
therapy."