Blog#244- 5/19/25
THE PROMISES AND PERILS OF GENE EDITING
By
Richard Davis
It would have seemed impossible only a few years ago, but medical researchers have been able to edit a baby’s genes so that he does not suffer from a rare genetic disease. A press release from the Children’s Hospital of Philadelphia explains, “In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine. The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. After spending the first several months of his life in the hospital, on a very restrictive diet, KJ received the first dose of his bespoke therapy in February 2025 between six and seven months of age. The treatment was administered safely, and he is now growing well and thriving.”
There have also been cases of gene editing for sickle cell disease and beta thalassemia. But in KJ’s case this is the first time gene editing was done for one individual which means the editing only applies to his case. For those other diseases, anyone who has them can use the same editing process.
All of these breakthroughs are possible because of the development of CRISPR (clustered regularly interspaced short palindromic repeats). The simple explanation of how this works is that researchers are able to cut a section of the genetic code that contains an aberration that causes a specific disease and replace it with a healthy section of code.
Research in this area has been going on for a number of years and there are a lot of ethical questions that surround the use of this technology. Scientists around the world realize that this technology could be used to create a new form of life and that it could be used to manipulate all of the genes a person has.
This is where the threat of designer babies comes in. It would be possible using CRISPR to focus on genes for intelligence or height or hair color or most human traits and design a baby of a person’s choosing. That has not happened yet but a Chinese scientist was jailed a few years ago for editing the genes of twins so they would not be able to contract HIV. This was a step too far for everyone.
The question remains. What is too far? No one has determined that and it is possible for scientists to do unethical procedures in their labs. Hopefully, the scientific community has people who respect ethical constraints.
There is another area where CRISPR and other genetic manipulations are now being used to look into the possibility of re-creating extinct species of animals. The work is already stirring things up in the scientific community.
George Church, a giant in the genetic research field, is a Harvard scientist who is now working with others to possibly revive the wooly mammoth. The steps that it would take to get there are no less dramatic than the Jurassic Park movie. In order to make the mammoth they have to find mammoth DNA, which it seems they have procured. They then have to use this DNA to create a mammoth embryo and then implant that embryo.
They would have to implant the embryo in an elephant, the closest species to the mammoth. No one knows what the result would be if it was carried to term. It is also somewhat alarming to think about an animal that is alive now but belongs to a world that went extinct 10,000 years ago. The creature would have no relation to the current world and, in many people’s minds that would be a cruel thing to do to an animal. Church and his colleagues have not been told to stop their work yet.
