The gene-editing tool CRISPR is one of the biggest achievements in modern genetics and also one of the most variable. Explained as a pair of “molecular scissors,” the CRISPR system employs an enzyme called Cas9 that binds to focus on places on the genome and enters in the DNA. Scientists desired to use CRISPR’s scissors to change out genetic mutations that cause disease and substitute them with healthy DNA.
Scientists of the Salk institute say that they have evolved a safer method of a familiar gene editing technology. On Thursday, they demonstrated that it reverses illnesses in mice, including diabetes, muscular dystrophy and kidney disease in more than 50% cases.
The famous gene editing tool CRISPR-Cas9 is well-known for aiding scientists edit a factor of DNA more exactly and efficiently than previous works.
“We moved this technique one big step toward human therapy,” said Hsin-Kai Liao, a post-doctoral researcher at Salk and co-first author on the paper.
Alexis Komor, a biochemist at UC San Diego who was not a part with the work, agreed. “This is a really thorough in vivo study that begins to bridge the gap between using CRISPR-based tools in cells in a dish and using them translationally,” she said.
Researchers are against to creating such gaps in the DNA of living humans because there are growing concerns relating to unnecessary mutations generated by the breaks.
“Cutting DNA opens the door to introducing new mutations,” said Belmonte in a press statement. “That is something that is going to stay with us with CRISPR or any other tool we develop that cuts DNA. It is a major bottleneck in the field of genetics — the possibility that the cell, after the DNA is cut, may introduce harmful mistakes.”
Scientists are attempting to use CRISPR to give the cell stalking orders since at least 2013.
By changing genome expression in the subject mice’s DNA, scientists observed a drastic improvement in the human diseases that the mice were experiencing. This is a small but important step for CRISPR-Cas9, which many have loudly praised as the future event.
They were also capable to generate some liver cells to metamorphose into pancreatic-like cells, which produce insulin, to moderately save a mouse model of Type-1 diabetes.
The team also exhibited that they could restore muscle growth and function in mouse models of muscular dystrophy, a disease with a familiar gene mutation.
In lieu of trying to fix the mutated gene, the researchers developed the expression of genes in the same method as the mutated gene, disallowing the effect of the diseased gene.
“We are not fixing the gene; the mutation is still there,” Belmonte said.
“Instead, we are working on the epigenome and the mice recover the expression of other genes in the same pathway. That is enough to recover the muscle function of these mutant mice,” Belmonte added.
Researchers are continuing further studies to make sure safety, practicality and capability prior taking into account conducting it to a clinical environment.