7 Exciting Developments Achieved with Crispr Technology

Considered one of the century’s most important discoveries, the CRISPR/CAS9 genetic engineering technology offers great promise because it directly targets the causes of diseases. Many countries worldwide seek cures using next-generation gene-editing methods, most notably crispr . Let us explore these exciting developments.

1. Removing HIV from Cells with Crispr

HIV is the virus causing AIDS. It infects immune system cells and also causes mutations in other cells. When HIV enters the body, it begins mutating rapidly and drug resistance develops. The greatest challenge in AIDS treatment is the emergence of drug resistance.

In May 2017, Molecular Therapy published a study in which researchers used CRISPR/CAS9 to successfully remove viral DNA from cells. The study, tested in 3 different animal models, achieved a high success rate. Human clinical trials are planned to begin soon.

2. Correcting Vision Loss with Crispr

“Leber congenital amaurois,” a rare genetic disorder appearing particularly in childhood, causes vision loss. In this syndrome, 14 genes responsible for healthy eyes are damaged.

Cambridge and Massachusetts universities are trying to eliminate this disease using CRISPR/CAS9. Following successes in preclinical studies, at the end of 2017, human clinical trials were planned to begin.

3. Treating Malaria with Crispr

Malaria is a life-threatening disease spread by parasites. It is usually transmitted through the bite of an infected female mosquito. Once transmitted to humans, parasites settle in the liver, multiply and then begin destroying red blood cells. According to World Health Organization statistics published in 2015, within one year there were 212 million malaria cases and 428.000 deaths.

Researchers at Imperial College London are trying to reduce malaria-spreading mosquito populations with CRISPR/CAS9. They focus primarily on 2 methods: genetically modifying males to produce more male offspring, and reducing females’ reproductive ability. Researchers have identified 3 genes determining female reproductive capacity and are working to target them with CRISPR/CAS9.

4. Intervening in Diseases at the Embryonic Stage with Crispr

With developing technology, CRISPR experiments can now be conducted within a few weeks. Its rapid introduction into human studies has also brought concerns about CRISPR. Perhaps one of the most critical issues is the ability to edit genes in “human embryos.”

In February 2017, a statement from the United States National Academies of Sciences, Engineering and Medicine emphasized that human gene editing is appropriate only under certain conditions. It also stated that genetically modifying cells in sperm, eggs and embryos is acceptable only if used to treat disease.

Although no research using CRISPR/CAS9 in human embryos has been conducted in America, researchers in China have used CRISPR and Cas9 to “insert new genes into human embryos and remove disease-causing genes.” In March 2017, the study was published in Molecular Genetics and Genomics. The genetically modified embryos were not transferred into humans.

5. Treating Cancer with Crispr

We know that “gene” damage underlies cancer. CRISPR/CAS9 genetic technology is thought capable of providing highly effective cancer treatment. The world’s first crispr clinical trials began in cancer. Two approaches stand out for its use in cancer treatment.

1. Correcting cancer-causing gene mutations and eliminating the disease
2. Modifying immune system cells to attack cancer cells more effectively.

CRISPR/CAS9 cancer treatment studies have mainly concentrated in China because legal limits on gene editing are somewhat more flexible. However, studies have accelerated in other countries too. In our previous article on this subject , as discussed, the first clinical trials of cripr/cas9 in cancer treatment have begun. The first, starting in October 2016, involved lung cancer patients. In this study, immune cells taken from patients are modified using CRISPR/CAS9 to attack cancer cells more effectively. The second trial began in April 2017, enrolling patients with stage 4 nasopharyngeal, stomach and lymph cancers: 20 patients. In addition, CRISPR/Cas9 has entered more than 20 clinical trials involving different cancers, such as breast and cervical cancer.

Another clinical trial has also begun in the United States with 18 patients with different cancer types.

Another exciting development concerns cancers associated with HPV (Human Papilloma Virus), the main cause of cervical cancer. Researchers designed a gel containing the CRISPR/CAS9 system and applied it to the cervix. Viral DNA was successfully eliminated from HPV-infected cells without damaging normal cells, preventing those cells from becoming cancerous. Phase 1 human clinical trials have begun and participants are currently being recruited.

6. Treating Multidrug-Resistant Bacterial Infections with Crispr

Resistant bacteria are among the greatest problems encountered in hospitals. In particular, a bacterium called “Clostridium difficile” causes fatal infections.

Researchers are trying to target “superbugs” using CRISPR/CAS9. A CRISPR pill under development aims to make bacteria destroy themselves. CRISPR sends a designed genetic message to a bacterium, causing it to cut its own DNA and disappear. This method is still being tested in preclinical studies and is thought likely to replace antibiotics in the future.

7. Crispr in Pain Treatment

Although chronic pain has no genetic basis, CRISPR/CAS9 is being investigated for pain treatment. Under normal conditions, inflammation is the immune system’s signal to “repair damaged tissues.” Chronic inflammation produces the opposite effect: tissue damage and severe pain.

In March 2017, University of Utah researchers used CRISPR/CAS9 to successfully inhibit cells causing chronic inflammation. Although still preclinical, this achievement offers great promise. Tissue deterioration (abnormal scars), particularly after surgery, may be delayed. Accelerating wound healing could reduce surgeries performed to correct defective tissues.