Abstract: Gene therapy, potential to cure a wide range of genetic diseases, has been a hot issue for a long time. However, ethical discussions about it never fade away, and legislation is being improved to help regulate the future research.
On February 1, 2016, the Human Fertilisation and Embryology Authority (HFEA) of the UK announced the world’s first formal approval of the request for editing of human embryos by Kathy Niakan of the Francis Crick Institute in London.
Kathy planned to use the CRISPR technique to answer questions like what genetic faults cause some women to miscarry and what is crucial for a healthy embryo. In 2017, her lab published the first major study in Nature, demonstrating that the transcription factor Oct4 is essential for fetal development.
Gene may sound mysterious to most people, but there are many reasons to support this research. An important reason is that modifying human DNA is the key to finding treatments for many diseases, helping people stay healthy and young, and live longer.
“It is estimated that human DNA has approximately 12 million single-nucleotide polymorphisms and thousands of copy number variants, most of which are harmless.” Said a scientist in Creative Biolabs, a leading service provider of gene therapy, “however, genetic disorders sometimes occur due to mutations that alter or inhibit protein function. The focus of gene therapy is to correct these mutated or defective genes.”
So far, the therapeutic effect of gene therapy has been far away from the expected outcomes. However, this method has great potential, especially for the treatment of genetic diseases.
Potential as it is, it must be warned that there is an important watershed in modifying human genes: who is the target population for genetic modification. For patients, it is a cure for the disease. However, if it is applied to healthy people, it is all about creating "Superman" or "God", which might result in accelerated evolution and the social disorder after it.
Considering gene modification, almost all countries are still conservative or even against it. It is believed that the changes in the genetic sequences will be passed on to all cells in the individual. What’s more, these modified genes will also be part of the human gene pool, which will be extremely difficult to reverse.
Since it’s of great danger to let things drifts on, US authorities have been promoting the related low and order. Over the past 45 years, gene therapy oversight by the National Institutes of Health (NIH) and the US Food and Drug Administration (FDA) has evolved significantly. In 1974, the NIH established Recombinant DNA Advisory Committee (RAC) to oversee the protocols of gene therapy trials. The FDA began to regulate gene therapy products in 1984 and issued its first guidance document in 1991.
Now, there is no federal legislation that bans protocols or places restrictions on experiments that manipulate human DNA. However, federal control does exist. For example, in 2015, Congress passed a provision stating that the FDA must approve any human clinical trial that involves gene editing. The FDA will certainly reject human trials that modify the human lineage via germline edits.
Gene therapy development is just like a car. Accelerator and engine cannot work alone, and brake is also necessary. Ethics, management and law are the brakes to keep humans safe and sound on the car of gene editing. Perhaps at a certain social stage, this technology can be proven to be effective and safe.