Disease and Health Disorder Prevention
CRISPR-Cas9 offers emerging opportunities in diagnosing and treating infectious diseases caused by bacterial, protozoan, viral, and other parasites.
The CRISPR-Cas9 system has revolutionized the cancer research through genetic screening of cancer genes, identifying novel drug target, or biomarkers for improved cancer diagnostics and therapeutics.
The following diseases are something that scientists are on the verge of beating through CRISPR-Cas9 technology:
Trials are already underway in China. The treatment involves immune T cells extraction of the patient. Through CRISPR, these cells are modified to get rid of a specific gene that is responsible for encoding the PD-1 protein. Some tumors can bind to this protein through the immune cell’s surface and pass on instructions to not attack healthy cells. The modified cells are infused back into the patient, who is now capable of fighting off cancer cells without the need for any medication or chemotherapy.
The very first clinical trial in the US and Europe has taken place, with the aim of treating blood disorders like sickle cell disease and beta-thalassemia, which specifically affect the blood’s oxygen supply system. Bone marrow stem cells are harvested using CRISPR technology so that they can produce fetal hemoglobin – a natural oxygen-carrying protein.
Experiments are underway to use CRISPR-Cas9 for cutting directly into the HIV virus’s DNA. Through this clever approach, the virus can be attacked and overwhelmed in its inactive form, which is why current HIV therapies can be so ineffective.
Since majority of hereditary blindness stems from a specific kind of mutation, CRISPR-Cas9 can be used to not only target but also modify only that gene. With the right CRISPR therapy, we can target the most common mutation responsible for blindness, restoring light-sensitive cells’ function as long as the patient has not gone completely blind.
Even though the process could be potentially risky, scientists are working hard to tweak CRISPR gene editing procedures to treat Huntington’s disease, a neurodegenerative condition involving a strong genetic component which affects 7 people out of every 100,000 across western countries.
Beyond palliative care, there is no treatment currently available for this condition which causes the muscles to shrink and become weak.
Research in mice has proven quite promising in terms of using CRISPR-Cas9 gene editing to rectify several genetic mutations responsible for the condition.
The condition which causes serious respiratory problems and has the potential to reduce a person’s lifespan to around 40 years, may be treatable through CRISPR technology – which can edit the mutations causing cystic fibrosis within the CFTR gene. Researchers recently proved that CRISPR can be used in human lung cells derived from cystic fibrosis patients, to fix a common mutation associated with the diseases, although more research is needed.