CRISPR Genome Editing for Better Health and Longevity

Genome editing, or gene editing, is a group of technologies that enable scientists to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. CRISPR technology has demonstrated applicability, simplicity, and efficient gene-editing capability compared to previously developed gene-manipulation tools.

Key takeaways:
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    CRISPR – gene editing technology.
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    Practical approach of this technology is being studied in various human life fields.
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    Medical applications of CRISPR are promising: over 10 clinical trials are underway.
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    Positive outcomes in mice – manipulating genes using CRISPR could extend lifespan.

Emmanuelle Charpentier of the Max Planck Institute for Infection Biology, Jennifer Doudna of the University of California, Berkeley, and Virginijus Siksnys of Vilnius University will each receive a gold medal and share the $1 million that comes with the Kavli Prize in nanoscience for inventing CRISPR. It is still unclear who first invented this amazing technology. However, Emmanuelle Charpentier and Jennifer Doudna received the 2020 Nobel Prize in Chemistry for their work on CRISPR technology.

The controversies surrounding CRISPR technology

CRISPR technology is undergoing several clinical trials and, therefore, is still awaiting approval for medicinal use — the technology needs more scientific proof. In addition, safety regulations governing its use would also need to be established.

However, professor He Jiankui shocked the world’s scientists in November 2018 when he announced that his team at the Southern University of Science and Technology in Shenzhen had used the CRISPR gene-editing system to edit DNA in human embryos to make them less susceptible to HIV. The edits disrupt the genes that code for a protein that allows HIV to enter immune cells. He used the experimental technology because the baby’s mother had HIV. Scientists condemned He’s actions, saying that gene-editing technology was too premature to be used for reproductive purposes. He Jiankui was given an 18-month suspended prison sentence and fined 500,000 yuan — about 70,000 USD.

Application of CRISPR

Each year, the dengue, chikungunya, and Zika viruses infect millions worldwide. The mosquito is the principal culprit behind the transmission of these deadly diseases. So, scientists developed a CRISPR system that alters insect genes to generate flightless female and sterile male mosquitoes. Interestingly, the edited gene could affect generations of mosquitos and eliminate fertile mosquito populations in the wild.

The application of CRISPR in plants and agriculture is fairly widespread. For example, a group of scientists using CRISPR modified tomatoes that produce more aromatic flavors. Additionally, they contain twice as much lycopene, an antioxidant thought to have health benefits. It can also modify genes to grow gluten-free wheat or decaffeinated coffee beans. CRISPR editing can also potentially delete the allergen genes at the source effectively to benefit allergic individuals.

Scientists are awaiting medical CRISPR innovations. As a result, people suffering from various diseases and medical conditions could greatly benefit from CRISPR technology — even if they don’t know it yet.

For example, two children treated with gene-edited cells to kill their cancers are both doing well more than a year later. Both baby girls were given the experimental treatment as a last resort. However, therapeutic clinical trials are now underway in children and adults alike in the UK.

The world’s first attempt to use the CRISPR gene-editing tool to treat blindness has shown hints of success. Six people received therapy starting in March 2020 — two now better sense light — one of whom even navigated a maze in dim light. In the trial, six people with a rare and fatal condition called transthyretin amyloidosis received a single treatment with the gene-editing therapy. All experienced a drop in the level of a toxic protein associated with the disease. Of these, two patients had a reduced level of toxic protein by an average of 87%. There are even more application fields using CRISPR.

New CRISPR gene editing clinical studies are rapidly emerging in the following areas:

AntibacterialsE. Coli Infections
Urinary Tract Infections
Blood diseasesBeta-Thalassemia
Transfusion-Dependent Β-Thalassemia
Haemophilia B
Human Immunodeficiency Virus Infection, HIV
Sickle Cell Disease
CancerAcute Lymphoblastic Leukemia
Acute Myeloid Leukaemia
Advanced Hepatocellular Carcinoma
B Cell Malignancy
B-Cell Non-Hodgkin Lymphoma
Bladder Cancer
Castrate-Resistant Prostate Cancer
Prostate Cancer
Multiple Myeloma
Eye diseaseLeber Congenital Amaurosis
Metabolic disordersType 1 Diabetes
Other genetic conditionsFamilial Hypercholesterolemia

Longevity and CRISPR

CRISPR gene editing has more than doubled the lifespan of mice engineered to suffer from progeria — premature aging. It also greatly improved their overall health. Progeria is a rare condition caused by a mutation, which probably occurs in the parent’s testes or ovaries. The average lifespan of children with progeria is 14 years. In this experiment, a CRISPR injection boosted the median lifespan from 215 to 510 days — the mice also experienced far more activity.

Wang Wei led a scientific team and conducted a genome-wide CRISPR–based screen to identify genes that could affect cellular senescence — a cessation of cell division. They identified the KAT7 gene as a driver of senescence in human adult stem cells. Inactivation of KAT7 rejuvenated prematurely aging progeroid human cells and extended the lifespan of mice. Furthermore, removing KAT7 alleviated senescence models of cancer, UV, and oxidative stress. These findings indicate that KAT7 gene inactivation reduces cell division in diverse biological contexts.

These results provide a significant new understanding of how scientists may eventually be able to target molecular aging drivers in humans.


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