The Next Gene Engineering Revolution: Curing Genetic Disease and “Designer Babies” Using Crispr

Today The Independent has a
fascinating article on a new extremely precise gene editing
technique called Crispr. The enzyme
CAS9 derived from bacteria enables researchers to edit genomes at
will. The technique clearly has major implications for treating
genetic diseases in people. Current techniques can insert new genes
but often they land fairly randomly and disrupt other genes. The
new technique appears to be so accurate and so safe that it could
be used to correct genetic flaws in human embryos. As The
Independent explains:

A breakthrough in genetics – described as “jaw-dropping” by one
Nobel scientist – has created intense excitement among DNA experts
around the world who believe the discovery will transform their
ability to edit the genomes of all living organisms, including
humans.

The development has been hailed as a milestone in medical
science because it promises to revolutionise the study and
treatment of a range of diseases, from cancer and incurable viruses
to inherited genetic disorders such as sickle-cell anaemia and Down
syndrome.

For the first time, scientists are able to engineer any part of
the human genome with extreme precision using a revolutionary new
technique called Crispr, which has been likened to editing the
individual letters on any chosen page of an encyclopedia without
creating spelling mistakes. The landmark development means it is
now possible to make the most accurate and detailed alterations to
any specific position on the DNA of the 23 pairs of human
chromosomes without introducing unintended mutations or flaws,
scientists said.

The technique is so accurate that scientists believe it will
soon be used in gene-therapy trials on humans to treat incurable
viruses such as HIV or currently untreatable genetic disorders such
as Huntington’s disease. It might also be used controversially to
correct gene defects in human IVF embryos, scientists said….

In addition to engineering the genes of plants and animals,
which could accelerate the development of GM crops and livestock,
the Crispr technique dramatically “lowers the threshold” for
carrying out “germline” gene therapy on human IVF embryos, added
Professor [Craig] Mello [of the University of Massachusetts Medical
School and 2006 Nobelist for his discovery of RNA
interference]….

Germline gene therapy on sperm, eggs or embryos to eliminate
inherited diseases alters the DNA of all subsequent generations,
but fears over its safety, and the prospect of so-called “designer
babies”, has led to it being made illegal in Britain and many other
countries.

The new gene-editing technique could address many of the safety
concerns because it is so accurate. Some scientists now believe it
is only a matter of time before IVF doctors suggest that it could
be used to eliminate genetic diseases from affected families by
changing an embryo’s DNA before implanting it into the womb.

“If this new technique succeeds in allowing perfectly targeted
correction of abnormal genes, eliminating safety concerns, then the
exciting prospect is that treatments could be developed and applied
to the germline, ridding families and all their descendants of
devastating inherited disorders,” said Dagan Wells, an IVF
scientist at Oxford University.

“It would be difficult to argue against using it if it can be
shown to be as safe, reliable and effective as it appears to be.
Who would condemn a child to terrible suffering and perhaps an
early death when a therapy exists, capable of repairing the
problem?” Dr Wells said.

Here’s hoping that the Crispr technique lives up to the hype.
Now if we can only keep the bioethicists (“science is outrunning
our regulations”) from interfering.

The
whole article is worth reading.

H/T Marian Tupy.