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.