CRISPR, a tool for editing the genetic code in living organisms, was first discovered in 2007. Since then, the prospect of eradicating diseases by editing the human genome hangs on the edge of science fiction and reality.
Here are four incredible breakthroughs from CRISPR that have taken place this year.
Better precision with a little protein
“If we think about how CRISPR will be applied in the future, it is currently one of the most important issues in the field: delivery,” said Jennifer Doudna, who co-won the Nobel Prize in Chemistry with Emmanuelle Charpentier in October. CRISPR, told Genetic Engineering News.
Access to DNA in living cells was the biggest challenge for CRISPR. The most widely used CRISPR gene processing system uses a protein called Cas9, which is found naturally in bacteria and archaea. Their biological role is to fight viruses by destroying the virus’ DNA and cutting it out of their genomes.
However, scientists can use it as a DNA shear, which can be used to cut disease-causing mutations from any DNA.
The problem is that Cas9, as well as other proteins used in various CRISPR systems, are very bulky, making it difficult to access the smallest nooks and crannies of the human genome.
However, in July, Doudna and her University of California Berkeley team announced that they were finding a new Cas protein.
Drumroll, please. Enter the small CasΦ (pronounced “Cas-phi”).
Nearly half the size, CasΦ can reach regions of the human genome that most CRISPR gene processing proteins cannot do. This new protein could be a game changer for genetic engineering.
Gene therapy seen as a drug for disease cells
This year, researchers announced the results of an ongoing clinical trial that tested CRISPR’s ability to modify the DNA of living cells and possibly cure genetic diseases in humans.
The study focused on two of the most common genetic disorders worldwide – sickle cell disease and beta-thalassemia. The only cure for these blood diseases is through a stem cell transplant from a viable donor. But often the recipients have to take immunosuppressants for months or years, with severe side effects.
The trials involved collecting blood stem cells from the patients. Thereafter, researchers used CRISPR to alter the cells’ genes and eventually transplanted the stem cells back into the patients. A few months later, when patients reported that their symptoms had disappeared, the scientific community viewed the study as a breakthrough through gene therapy and called it a cure.
“I am encouraged by the preliminary results, which essentially show a functional cure for patients with beta-thalassemia and sickle cell disease,” researcher Haydar Frangoul said in a news release.
The next step is to expand the study by enrolling and observing 45 patients in the long term.
Genetically modified pigs can be future organ donors
The US does not have enough donor organs to meet every need. As a result, about 17 people die every day because they do not get the liver, heart or lung they need.
To solve this shortage, scientists have been donating cross-species donations for ten years – or xenotransplantation – with little progress. Our immune system is designed to attack invaders, which is great if you have the flu, but it makes xenotransplantation virtually impossible.
“The approach, if validated by further studies, could help address the global organ shortage (and) alleviate transplantation needs.”
Thanks to CRISPR, xenotransplantation may be possible. Using CRISPR, researchers from Qihan Biotech adapted the DNA of pigs to make them more compatible with humans. The researchers applied 13 pigs to the pigs, all in the hope of making them more acceptable to the human body. In vitro laboratory tests have shown promise – cells from the ungroomed pigs appear to be less likely to be rejected by the human immune system than those of unmodified animals.
“The approach, if validated by further studies, could help resolve global organ shortages (and) alleviate transplantation needs,” said George Church, co-founder of Qihan Biotech, in a press release.
Further studies are underway: the team is now testing an organ transplant from a pig that is cultivated by no one in a primate.
CRISPR enters human body for the first time
Scientists from the Casey Eye Institute in Portland used CRISPR for the first time in the human body – in a patient with an inherited form of blindness.
Doctors dropped the gene editing tool behind the retina via three drops of fluid through a tube the size of a human hair. Once in the body, CRISPR went to work. It cut the mutated gene on both sides of the problem area. They hope that once the mutation is removed, the cut DNA will reconnect itself so that the gene can function as it should. They are now in clinical trials.
“Once the cell is edited, it is permanent and will hopefully last the cell for the life of the patient,” because these cells are not divided, Dr. Eric Pierce of Massachusetts Eye and one supervisor said who was not involved in this first case. Over.
If all goes according to plan, the patient should regain his sight within a few months. The doctors then plan to further test the procedure on 18 children and adults.
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