Rats engineered to have a genetic form of blindness, known as retinitis pigmentosa, have had their vision partially restored by gene therapy.
The therapy involves using a gene editing tool known as Crispr – this works by snipping out strands of DNA and replacing them with healthy versions. This tool is often described as a ‘molecular scissor’. This is done by injecting a virus carrying a package of gene-editing instructions into the eye of the rats. Tests done on the rats after 8 weeks showed that their retinal cells had begun to heal, and their eyes could respond to light
Previously, Crispr was only able to work on cells that divide, such as embryonic cells. However, a latest breakthrough means that scientist can now use this type of gene therapy on adult cells that don’t divide, such as those in the eye.
This technique could be trialled in humans within the next 2 years, and could potentially be used in illnesses such as cystic fibrosis, muscular dystrophy, and haemophilia. Other ways that Crispr could be used include making immune cells resistant to the HIV virus so that it cannot take over the body, even if it gets into the blood.
However, this does raise questions about the ethics of genome testing – would it be right to ‘edit’ human embryos? And what limits should we impose on ourselves? Could we end up with designer babies? I think this is an important discussion that needs to take place as this technology develops.
Interestingly, the NIH (National Institute of Health, a government organisation) does not fund any gene editing on human embryos. However, if this new piece of research leads to success in clinical trials, will the NIH fund it, particularly if it can cure diseases that currently have no treatment?
Another interesting piece of information I found was the fact that there are no laws against editing the human genome in the USA. Perhaps it is a matter of where, not if, research will take place into editing embryos.
Thank you for reading.
-Idil
Sources:
Genome editing: how to modify genetic faults
thesciencereport 