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How Safe Human Genome Editing Could Benefit Everyone

Human Genome EditingAfter two years of study, the World Health Organization (WHO) released two reports on how to use human genome editing safely and inclusively. The editing technology has significant potential to cure many diseases but the drawbacks must be considered, experts say. Human genome editing safety remains a priority and ensuring everyone has access to the technology could lead to significant improvements in the fight against poverty. The process warrants significant attention: It could further inequality but could also be a massive step toward eradicating poverty.

What is Human Genome Editing?

Somatic and germline editing are two primary types of genome editing. Somatic editing is surface-level and can be used to treat a disease with genetic origins. For example, a scientist can take a patient’s blood cells and utilize CRISPR technology “to edit blood cells as a treatment” for “blood disorders.” This genome editing type does not get passed down to any children.

Germline editing, the more controversial type, changes the genome of a human embryo at the earliest possible stage. It impacts all cells, which could affect any children one has in the future. Although germline editing raises significant ethical questions, it does have the potential to prevent several diseases from manifesting in a child. Currently, international policies limit germline editing, sometimes allowing it for only research purposes. If an individual utilizes edited embryos to “initiate a pregnancy,” this would be considered heritable genome editing.

Heritable genome editing makes changes to the “genetic material of eggs, sperm or any cells that lead to their development,” which includes early embryos. Human genome editing safety sparks serious ethical and controversial concerns, thus, restrictions and guidelines exist worldwide.

Considering the Positives

The potential to cure serious diseases is enormous despite ethical questions. Faster diagnoses, accurate treatments and disorder prevention efforts all could be achieved or improved through genome editing, according to the U.N. In fact, somatic gene therapy made significant strides toward treating HIV and sickle-cell disease in recent years.

Fertility and disease resistance could both improve with the technology’s use. Human genome editing can and already is a way to treat or prevent many serious diseases, and overall, improve life for many. If used correctly, in a safe and efficient manner, the entire world could benefit.

Considering the Negatives

The potential is enormous, but so are the risks. Political and social justice issues are very important to consider, especially when it comes to germline and heritable genome editing. Editing could affect the very issues movements fighting for a broad range of social and economic issues raise.

Germline and heritable human genome editing both have ethical and moral questions. There is a possibility the genetic changes can be passed down to future children. It could be used as a way to improve traits in an irresponsible manner and access could vary for many.

Somatic editing also faces challenges. Rogue clinics and “illegal, unregistered, unethical or unsafe research” pose serious threats. Also of concern are “activities including the offer of unproven so-called therapeutic interventions.” Human genome editing safety is a difficult but important task to undertake as the treatment could be harmful if used incorrectly.

Another serious issue to consider is who would receive the treatment. This could just further the medical inequality divide between wealthy and lower-income nations as the treatment is expensive. As many nations with fewer resources have more difficulties with diseases, the treatment will be especially beneficial for them. However, these nations might not have effective access.

How Genome Editing May Help Those in Poverty

Diseases that tend to affect those in poverty because of a lack of treatment could be treated with human genome editing. These include diabetes, alcohol-attributed diseases, malaria and others. Improved treatment from human genome editing could lead to significant strides in reducing poverty. For those with the least access to or possession of societal resources, editing could potentially be a benefit. Other diseases like “cystic fibrosis, cancers, muscular dystrophy and Huntington’s disease” could also be treated or cured.

If properly managed, the impact of human genome editing on those in poverty could be significant, increasing health across the board. If recommendations from the WHO are properly followed and scientific progress continues, the benefits for the global population could balance the risks.

– Alex Alfano
Photo: Flickr

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CRISPR Gene Editing To Treat Sickle Cell Disease

Treat Sickle Cell DiseaseCRISPR gene-editing technology is now being used to treat various illnesses. This holds the potential to be a life-changing development for many people and may treat those plagued with sickle cell disease around the world.

What is Sickle Cell Disease?

Sickle cell disease is most prevalent in African countries, where having one copy of the sickle cell gene helps protect people against malaria. However, having two copies of this gene results in sickle cell disease. Sickle cell disease occurs because of a genetic mutation that causes red blood cells to develop a sickle-shape and this obstructs healthy blood flow. The condition can cause serious pain and negative health effects, usually resulting in early death. When considering children with the disease, 70% are born in sub-Saharan Africa. Unfortunately, these countries do not have adequate resources to properly alleviate the symptoms of this condition, let alone treat them.

A Potential Cure

In recent months, it has been discovered that CRISPR gene-editing technology may be the key to curing sickle cell disease. CRISPR–Cas9 is a naturally occurring defense system that edits DNA sequences to fight viruses in the human body. In the past decade, scientists have discovered how to harness this system’s ability to manipulate DNA in chosen ways. The result of this is CRISPR gene editing is a powerful technology that can correct genome defects and even alter entire genomes.

CRISPR technology works by editing genes, which modifies how the body functions. First, medical professionals remove patients’ bone marrow and treat it. Then, CRISPR allows scientists to “cut and paste” bits of the genome by either cutting or adding a sequence of DNA into the genome. This can correct genetic mutations, ultimately improving a patient’s health.

In the U.S., a trial of using CRISPR to cure sickle cell disease is yielding promising results. The treatment uses CRISPR technology to activate a gene that instructs the body to produce fetal hemoglobin instead of adult hemoglobin. The presence of fetal hemoglobin prevents the blood cells from sickling. In this way, the treatment alleviates the health complications typically resulting from sickle cell disease. The subject of this trial is much healthier and has made exceptional progress in her recovery. These spectacular results have left many people hopeful that CRISPR technology could successfully treat sickle cell disease, with more widespread results by 2022.

The Future of CRISPR Treatment

For CRISPR treatment to reach its full potential, it must become more accessible to those who need it most. Therefore, the underprivileged in sub-Saharan Africa would benefit greatly. One suggested way to overcome accessibility barriers is through a tiered-pricing system. This system would offer gene therapy treatment to patients in developing countries at a reduced price, while patients in high-income countries would be expected to pay for the treatment in full.

There are currently logistical barriers to this solution, as gene therapy can cost thousands of dollars. The cost of CRISPR treatment would have to be greatly reduced (beyond the normal price drops of tiered pricing) to be successfully made available to the underprivileged. Additionally, this treatment requires consistent doctor visits. Much of sub-Saharan Africa lacks access to health clinics and other essential resources, such as refrigeration.

Breaking Down Barriers

Organizations are helping to eliminate the barriers blocking CRISPR treatment for sickle cell disease in developing countries. The National Institutes of Health and the Bill and Melinda Gates Foundation donated $200 million to this cause in 2019. This money will help make gene therapy accessible throughout the world and improve the quality of life for thousands. With the promise of affordable CRISPR gene modification therapy, there is hope for individuals worldwide to treat sickle cell disease. Permanently improving the quality of life is the end goal. Those living in developing countries, the global poor and those vulnerable to falling into poverty will be the most to benefit from this exciting, technological development.

– Hannah Allbery
Photo: Flickr