On the brink of a biomedical revolution

February 12, 2020

Gene editing might give humans the tools to write diseases out of our DNA while refining medical research and diagnostics, but with incalculable risks to ponder


On the brink of a biomedical revolution

 

Medical journalism lends itself to sensationalism. News portals are often peppered with eye-catching headlines on breakthrough drugs and technologies and the miraculous health powers of fruits or vegetables. Journalists, after all, need to sell their stories, and medical research is a hotbed of discoveries and inventions with great potential but illusive rewards. That’s just part of the trial-and-error of scientific research, which often requires failure before success. 

Good health journalists will avoid the hype around the latest innovations, while highlighting the considerable time needed to measure their real impact on patients and doctors. But there are some rare cases when even the most hardened objective journalists can’t help hailing a new innovation as a potential game changer. One of these is gene editing, and CRISPR in particular.

A technique for the manipulation of DNA, the basic code of every living organism, gene editing offers the chance to modify the instructions driving cellular processes across the entire biological world. Put in more vivid terms, gene editing might engineer plants with desirable characteristics like resistance to drought and pests, create mosquitoes unable to carry deadly diseases such as malaria, and even lead to cures for devastating genetic conditions by fixing their root causes, the underlying mutations in genes.

None of this is breaking news, though. Scientists have been manipulating the genome of plants, animals, and even humans for decades. The process, however, has gained momentum with the introduction of CRISPR. This latest approach, widely considered the easiest and cheapest form of gene editing, involves the chemical engineering of molecules mimicking those naturally present in a segment of bacterial genome.

“I think that if you apply the CRISPR technology correctly, this method will revolutionise the entire biomedical field and change the way we treat patients with infectious and genetic diseases as well as with cancer because you can use CRISPR to correct mutated genes,” Dr Kamel Khalili, director of the center for neurovirology and the Comprehensive Neuro AIDS Center at Temple University’s Lewis Katz School of Medicine, told Global Health Asia-Pacific. He’s currently researching the use of CRISPR to wipe out the human immunodeficiency virus (HIV) in patients infected with the as-yet-incurable disease.

And this is just the start. Down the road, CRISPR might even allow scientists to make non-medical enhancements in the genetic code of embryos, providing future generations with genes to make them taller or more athletic — a prospect dreaded by many as a dangerous new form of eugenics.

What this means is that we may now be on the brink of a seismic revolution, one that will usher in a new era where humanity plays an active role in directing the evolution of life and its own genetic future in particular. In the biomedical field, it’s hard to imagine a more consequential revolution than the one gene editing is likely to bring about.

 

The advent of CRISPR

Accumulated in the bacteria’s genome as part of an evolving immune system, CRISPR (short for ‘clustered regularly interspaced short palindromic repeats) contains bits of viral DNA the bacteria have snipped from invading viruses and stored for future immune response. When viruses of the same species infect the bacteria again, CRISPR produces molecules that hunt down and stick to the invaders while a scissor-like protein called Cas9 shreds their DNA, killing the viruses.

Essentially, CRISPR collects mug shots that allow the bacteria’s immune system to flag harmful viruses that need to be eliminated by proteins instructed to cut up specific sequences of DNA.     

“It was the perfect bacterial weapon: a virus-seeking missile that could strike quickly and with incredible precision,” Dr Jennifer Doudna, Professor in the department of Molecular and Cell Biology at UC Berkeley and a CRISPR pioneer, wrote in A crack in creation.

In a 2012 landmark experiment, Dr Doudna and other researchers managed to reprogramme CRISPR and Cas9 to pinpoint and cut specific sequences within the DNA of a jellyfish while repeating the same feat with the human gene CLTA in a subsequent study. Coupled with cells’ ability to reattach DNA strands that have been sliced apart, CRISPR-Cas9 proved effective at editing the key biological instructions regulating the development of all living organisms.

“With that success, we had validated our new technology that offered scientists the remarkable ability to rewrite the code of life with surgical precision and astonishing simplicity,” she wrote.

Since its introduction, CRISPR has already been used to edit the genome of numerous plants, including tomatoes, oranges, rice, and wheat and animals like mosquitoes, mice, and monkeys as well as a variety of human cells.

CRISPR, however, is just the most recent gene-editing technique. Previous technologies, such as zinc-finger nucleases (ZFN) and transcription activator-like effector nucleases (TALEN), have achieved remarkable results but, according to most experts, are much more complex and costly than CRISPR.

Before Dr Doudna’s team made their experiment with human genetic material, other researchers managed to edit CLTA by using ZFN. It took them months to design the ZFN tool and its cost at the time stood at US$25,000, she explained. In comparison, her team spent minutes to design the analogous version of CRISPR that ended up editing CLTA, with a price tag of a few tens of dollars.

“CRISPR finally made gene editing available to all scientists. Previous tools —primarily ZFNs and TALENs — were difficult to design and prohibitively expensive. For this reason, many labs, including my own, were unwilling to take on the challenges of research using gene editing,” she wrote. “Some experts have suggested that, with today’s tool, anyone can set up a CRISPR lab for just US$2,000.”

The report, titled The CRISPR revolution: changing life, by The Royal Society in the UK sounded a similar note, highlighting that CRISPR is “faster, cheaper and more accurate” than other gene-editing techniques and it has already been applied to “a wide range of organisms, giving researchers a powerful tool to use in previously difficult or impossible scenarios.”

Though some argue CRISPR will have its most significant impact in engineering better crops and animals for human consumption, it’s hard to overestimate its potential applications in the clinic.  

“If scientists could safely and efficiently deliver CRISPR into the human body so that gene editing worked as well in patients as it did in lab-cultured cells, then the possibilities for transforming medicine would be boundless,” wrote Dr Doudna.

But the technique is not perfect. A major problem associated with it is its off-target cuts within the DNA. These are random and unintended changes CRISPR makes to sections of the genome other than those it’s supposed to achieve. “CRISPR could make occasional mistakes and confuse one letter of DNA for another,” she acknowledged.

Last year, one study suggested CRISPR is responsible for much greater DNA damage than previously thought. “This is the first systemic assessment of unexpected events resulting from CRISPR/Cas9 editing in therapeutically relevant cells, and we found that changes in the DNA have been seriously underestimated before now. It is important that anyone thinking of using this technology for gene therapy proceeds with caution and looks very carefully to check for possible harmful effects,” Professor Allan Bradley, a researcher at Wellcome Sanger Institute and one of the authors of the study, said in a press release.

It’s still unclear what impact off-target changes could have on health, if anything. The human genome, in fact, is in a constant state of mutation due to external factors like radiation or internal random mistakes in DNA replication, with everyone experiencing about one million mutations per second.

With researchers already working towards solving the problem, Dr Doudna is confident CRISPR will eventually achieve a sufficient level of accuracy and safety to enable use in the clinic.

 

A straightforward application: Curing genetic diseases

Genetic diseases are the natural target of gene editing. Driven by abnormal mutations in the genome, they are often incurable because their root cause can’t be fixed, until now.

Among them, monogenic conditions, or those caused by abnormalities in one single gene, are the easiest to tackle through gene editing because they’d only require changes to one specific section of the genome while other genetic diseases are driven by multiple mutations in different genes, making the editing more complex.

Some devastating monogenic diseases CRISPR might help cure include blood disorders, such as sickle cell disease (SCD) and thalassaemia, cystic fibrosis, which affects the respiratory, digestive, and reproductive systems, and Huntington’s disease, a brain disorder leading to cognitive and movement disorders.

Research on the efficacy of gene editing to cure SCD and thalassaemia is particularly advanced, with some companies already running clinical trials testing treatments on patients.

Both conditions harbour genetic mutations that disrupt the functions of haemoglobin, a protein responsible for transporting oxygen in the blood. While thalassaemia is characterised by insufficient production of haemoglobin, leading patients to anaemia (tiredness and shortness of breath), SCD produces sickle-like red blood cells that tend to clump together, thus clogging up blood vessels.

“SCD is a cruel disease that leads to the deterioration of the functions in several organs like the lungs, kidneys, and heart, cognitive problems and debilitating pain that can be recurrent or chronic,” Dr Daniel E. Bauer, a paediatric haematologist at Boston Children’s Hospital, told Global Health Asia-Pacific. “It can be associated with short lifespans. In the US, patients usually reach their 40s, but in many other countries they die during childhood.”

There is some good news for SCD sufferers, however. The complexities of human biology, together with a rare and harmless condition known as hereditary persistence of foetal haemoglobin (HPFH), are guiding researchers like Dr Bauer towards a potential cure for SCD and thalassaemia. While foetal haemoglobin allows oxygen transportation in unborn babies, its production is normally shut down soon after birth, when another type of the protein, called adult haemoglobin, takes over. As implied in its name, people with HPFH continue to have high levels of foetal haemoglobin in their blood. This makes no difference for healthy individuals, but it dramatically changes the life of SCD patients.

“People with SCD and thalassaemia who have increased expression of foetal haemoglobin have milder diseases because the alternative haemoglobin can replace the defective adult one,” explained Dr Bauer, noting that this observation pushed him to explore whether gene editing could reactivate the production of the foetal protein.

With the gene BCL11A controlling the switch from foetal to adult haemoglobin, Dr Bauer’s team designed special CRISPR-Cas9 molecules to disrupt key sequences of the gene in the blood stem cells harvested from patients with SCD and beta thalassaemia. Tasked with churning out all the components in the blood, stem cells are to be fixed if we want to provide patients with a constant stream of healthy blood cells.

The stem cells with modified BCL11A were then injected into mice, where they gave rise to perfectly functional blood cells having high levels of foetal haemoglobin but no unwanted side effects.

“Given the success we have observed so far we are very optimistic to initiate a clinical trial where we can test the safety and feasibility of this method in actual patients with the goal of giving rise to durable modified blood-forming cells that improve effective haemoglobin expression,” said Dr Bauer. “What we are doing is harnessing a natural mechanism to improve the diseases.”

Given it’s potential, there’s naturally a lot of excitement now around the use of gene editing to correct haemoglobin disorders, with many research teams applying different methods to achieve the same goal of a permanent fix.

Like Dr Bauer’s team, Sangamo Therapeutics, a US company in the field of genomic therapies, is attempting to design a gene-editing treatment for haemoglobin disorders by increasing the levels of foetal haemoglobin in the blood — the only difference is the editing tool, ZFNs instead of CRISPR-Cas9.

Using a different strategy, other researchers aim to edit the faulty gene driving the disorders in the hopes of turning on the production of functional adult haemoglobin.

In 2017, one teenager with a severe form of SCD was cured after receiving a healthy version of the adult haemoglobin gene that instructed his stem cells to form normal oxygen-carrying proteins in a treatment known as gene therapy. Unlike gene editing, this approach doesn’t involve any change to the original genome of the patients.

“Like many things in medicine, we’ll have to compare the different approaches to determine which is the most effective, the safest, and the one that can be accessed by the largest number of patients,” he said.

 

Uprooting HIV: Can gene editing delete viruses?

In a clear sign of its remarkable curative potential, gene editing might mark a step change in the fight against viral diseases as well, with HIV researchers leading the way.

“When human cells get infected with HIV, the viral genome gets incorporated in the cellular DNA,” Dr Khalili.

This trick played by HIV explains why researchers are struggling to find a definitive cure for it, despite the mind-blowing advancements made over the last decades in the management of the once-deadly infection. Antiretroviral (ARV) medications, in fact, are pretty good at keeping HIV in check, so much so that patients who take them are protected from developing AIDS and can’t even transmit the virus to their sexual partners. If they stop taking the medications, however, the virus embedded in their DNA begins to replicate again, forcing patients to stay on ARV for life, which exposes them to a host of side effects, including neurological problems, kidney disorders, and bone disease.

With this obstacle in mind, “we started looking at HIV as a genetic disease instead of a simple infectious disease and focused on utilising a genetic strategy, CRISPR technology in particular, for achieving the elimination of the viral DNA in the patients’ genome and hopefully a cure for it,” explained Dr Khalili.

The first experiment involved testing the efficacy of CRISPR-Cas9 in eliminating HIV in cultured cells in Petri dishes. This ended in a success that provided proof of concept for the validity of the treatment.

To demonstrate that the therapy could snip HIV out of cells inside living organisms, Dr Khalili’s team infected 23 mice with human cells carrying HIV DNA. Once the virus started replicating, the animals were first treated with ARV drugs to suppress viral replication and subsequently injected with CRISPR-Cas9. ARV was then discontinued, and nine mice had no virus re-bounce.

“We looked for the virus in every tissue and cell and we found none. This means CRISPR-Cas9 was able to completely inactivate HIV so as to prevent its replication,” said Dr Khalili. “In the rest of the animals the virus was still present, but I think the approach needs to be refined to completely eliminate the virus in all the models. For instance, we should probably increase the number of injections.”

In order to refine the therapy, the team is already setting up a study on larger animals, non-human primates, that give more solid indication than mice of how the treatment might affect humans. If the positive results hold up, this will pave the way for testing the approach on patients in a clinical trial.

“To my knowledge, this is the first study demonstrating that HIV can be eliminated in animal models. Hopefully, this treatment can be perfected for the clinic, but we are not there yet,” he said.

In a similar attempt to cure HIV with gene editing, other researchers are targeting a human gene involved in HIV infection, called CCR5.

In order to infect human cells, one of the more common forms of HIV has to latch on to a protein coded by the CCR5 gene, so people without that protein due to a naturally occurring mutation in the CCR5 cannot get infected with that strain of the virus. As a testament to the power of this benign glitch, the only two HIV patients who have been cured so far received blood stem cell transplants from donors with mutated CCR5.

Sangamo Therapeutics, for instance, is running a trial where CCR5 is edited in patients’ immune cells through ZFNs to test whether this will provide resistance to HIV. 

Despite the promise of editing CCR5, Dr Khalili believes targeting HIV directly, instead of tampering with human genes, is the best way of harnessing gene editing against HIV.

“I don’t know if our approach is more promising than editing the gene CCR5, we just have to wait and see,” acknowledged Dr Khalili. “But I think our approach is straightforward, we are targeting the viral gene and we are trying to avoid the manipulation of the cellular gene. CRISPR is still a young technology so it may be too early to embark on that strategy.”

If successful, Dr Khalili’s approach might also open the door for editing other viruses off the human genome, leading to a cure for multiple viral diseases.

“I think CRISPR is a powerful technology for the treatment of a number of infectious diseases,” said Dr Khalili. “We have already published a paper showing that herpes simplex virus can be successfully edited in cultured cells in a way that leads to its suppression.”

Several other viral conditions might be next in line for a CRISPR cure.

 

The ethical implications of germline editing

In a heatedly debated case that pushed what many perceive as the ethical boundaries of medical research, last year the Chinese scientist He Jiankui announced he had used CRISPR to edit CCR5 in two embryos that later gave rise to a pair of baby twins — a practice called germline gene editing.

Though He’s objective was to avoid transmission of the virus from the HIV-infected father to the newborns, his experiment drew widespread outrage for exposing the babies to potentially high risk with no benefits.

Echoing the opinion of the scientific community at large, the World Health Organization (WHO) said “it would be irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing,” according to a statement put out last March.

Unlike the somatic gene editing approaches pursued by Drs Bauer and Khalili and Sangamo Therapeutics, germline gene editing involves changes to the genes of germline cells, such as egg and sperm cells or embryos, that in turn will be passed down to all future cells derived from the original edited one. In the case of He’s experiment, this means the pair of baby twins, along with all future generations born from them, will have a mutated CCR5 gene in every single cell making up their bodies. In stark contrast, patients who undergo somatic gene editing will only see modifications in a number of their own cells and won’t transmit those changes to their offspring.  

“He has exposed the babies to potential multiple risks without any real gain,” Dr Tamra Lysaght, assistant professor at the Centre for Biomedical Ethics of the National University of Singapore, told Global Health Asia-Pacific. With every gene playing a role in the expression of multiple proteins involved in different biological processes, editing one gene might lead to unknown unintended consequences.

“As we don’t know all the proteins CCR5 codes for, its editing could lead to massive risks for the babies,” she said.

Demolishing He’s rationale for the editing, Professor Lysaght pointed out that the experiment was not justified given that there are effective and safer alternatives to avoid HIV transmission in newborns. “Fertility clinics have standard procedures like pre-implantation genetic testing to ensure that embryos do not get exposed to HIV.”

On top of that, there are big ethical dilemmas that should give pause to the supporters of germline editing.

Unlike somatic gene editing, modifying the genes in the germ line “is not ethically acceptable because the changes are passed from one generation to another and those generations have no say whatsoever in the manipulation of the genome,” stressed Professor Lysaght.

Sketching out an even more worrying scenario, some critics of germline gene editing warn that sanctioning the technology would open a Pandora’s box of genetic enhancements to design the perfect baby, not only better protected from diseases but also with desired physical and mental characteristics, from eye colour to intelligence.

“Once you start to design your child and say ‘I want this characteristic and I don’t want that characteristic,’ you are turning your child into just another design commodity that you would buy on the market. For example, when you go to buy a refrigerator you ask for the size you want, the colour and other specifications,” Dr David King, founder of the advocacy group Human Genetics Alert, said in an interview with Global Health Asia-Pacific.

In his view, this will also compound existing health inequities as financial resources will allow the well-off to give their children a genetic edge over the rest — an advantage that will be passed down to future generations, further amplifying the distance among social classes. 

“My concern is that what you’ll do by allowing to modify your children’s genes is to create a society in which some people’s children will be given extra built-in advantages over other people’s children,” he said. “And this will radically exacerbate the social inequalities that are a major problem in our societies already.”

Though Dr Doudna referred to He’s experiment as ‘horrifying’ and hoped stringent regulatory guidelines would be put in place, during a talk at the University of California in Berkeley, she said that germline gene editing was likely to move forward and gain acceptance over time in the same way as in vitro fertilisation did.

“Now, does that mean that we’re entering into an era of eugenics? I don’t really see that likely to be happening. I think that it’s probably going to be more sporadically utilised, and I would hope that initial uses are limited to real medical need rather than what we might consider to be enhancements,” she said.

However, the number of cases where editing germ cells or embryos will be medically useful is negligible, according to Professor Lysaght, as alternative options available in fertility clinics already allow most patients to avoid the implantation of embryos with genetic abnormalities.

“Supporters of germline gene editing will say that this will help parents with debilitating genetic diseases to have children without those diseases. Unfortunately, I think that that market is so miniscule that that’s not the reason why the technology is going to get developed,” she said. “Instead, it will be developed to get into the much more lucrative market of genetic enhancement.”

  

The diagnostic power of CRISPR

A less controversial CRISPR application aims to turn around the detection of infectious diseases like dengue, Zika, Ebola, and many others, in a way that doesn’t require any editing of human cells and hence doesn’t raise strong safety concerns. 

Standard tests to diagnose those conditions require time, sophisticated equipment, and skilled personnel — a mix of assets that is challenging and costly to put together, especially in under-resourced areas.

“We need better diagnostic tests for haemorrhagic fevers, which include Lassa fever, Ebola, dengue fever, and Zika, because current tests take a long time, about four hours, and are not suitable for outbreaks that require quick, cheap, and accurate diagnostics,” Christian Happi, Professor of Molecular Biology and Genomics at Redeemer’s University in Nigeria, told Global Health Asia-Pacific.

Currently, the diagnosis of haemorrhagic fevers is done through polymerase chain reaction tests that require a laboratory staffed with technicians specifically trained to perform the examination.

But this might change thanks to a CRISPR-based technology developed by researchers at the Broad Institute in the US that promises to dramatically simplify and speed up the diagnostic process.

Combining two methods called SHERLOCK and HUDSON, the technique makes use of the enzyme Cas13 — another CRISPR-associated protein similar to Cas9 but able to cut RNA instead of DNA — paired with a GPS-like molecule that can be programmed to guide the enzyme to specific RNA sequences, like those making up the genome of viral diseases. Unlike Cas9, though, Cas13 not only cuts its intended target but starts slicing other RNA molecules indiscriminately. By adding a reporter RNA molecule that changes its colour once cut, the system makes for a quick and inexpensive paper-based test that has already shown promise in detecting dengue in samples of saliva and blood.

If the system could be successfully deployed in the field, it would be the equivalent of a pregnancy test for infectious diseases, according to Professor Happi, cutting diagnosing time to about one hour and a half or less and allowing primary care physicians in rural areas to diagnose the diseases without any sophisticated equipment, highly-trained manpower, or electricity.

The professor is collaborating with researchers at the Broad Institute to adapt the system to diagnose Lassa fever, a viral condition that is endemic in many countries in West Africa, where it claims about 5,000 lives every year, according to him.

By using SHERLOCK and HUDSON, “we have been able to diagnose the presence of Lassa in the blood, now we are trying to do the same in urine and saliva samples to develop a simple technology, which is very important in poor-resourced settings like ours,” he said.

Not only would a diagnosis done with saliva or urine samples avoid the need for blood collection, but it would also decrease risk for healthcare professionals. “When you collect blood from patients with deadly diseases like Lassa or Ebola, you are exposing yourself to them, increasing the risk of infection, while saliva and urine samples reduce the risk of infection for medical personnel.”

Time is of the essence when dealing with highly contagious and potentially lethal diseases like Lassa, and a quicker test will translate into better infection control within hospitals and more favourable prognoses.

“This would change the diagnostics of the disease tremendously. Patients usually present at the hospital with symptoms that cut across many diseases. For instance, many symptoms of Lassa fever and Ebola are very similar to those of malaria and other diseases.

“So, how do you differentiate between them in a very short time so that the clinician will know the course to follow? How do you triage and separate patients in a way that can reduce disease transmission in medical facilities as early as possible? It’s possible the person with Lassa is contaminating other patients without knowing it. So, it’s important to have a quick method,” explained Professor Happi.

On top of that, the earlier Lassa is diagnosed the better. “If you come to the hospital with a Lassa fever infection that is beyond seven days, then the prognosis is poor. We are still doing more tests in the lab for validation and then we’ll move to deployment in the field,” said Professor Happi. “We are targeting the end of this year.”

Ultimately, the goal is to design a system that uses one single paper strip to diagnose all haemorrhagic fevers quickly without the need of electric-powered equipment and blood samples.

 

A research tool for cracking the genetic code of diseases

While waiting for its clinical applications to pass regulatory muster and bear fruits, CRISPR is already boosting medical research, with genomics and cancer as two key areas expected to benefit the most. 

Though we obtained a complete map of the human genome in 2003, when the Human Genome Project was completed, we still have a limited picture of the functions played by the genes, let alone their complex interaction.

“There’s a lot we don’t understand about how the human genome functions and how it supports normal health or causes diseases,” said Dr Bauer. “We have all this genetic data but often it’s hard to know which genes are really causing the problem.”

CRISPR is turning things around by permitting scientists to knock genes on and off so as to observe which biological traits are affected. This should widen knowledge on the normal functions of the genes as well as their role in the processes leading to diseases. “You can learn a tremendous amount of information about the biology of diseases and potential therapeutic options by being able to edit the genome,” he said.

One shining example is cancer, a disease rooted in genetic mutations that drive cells to grow in an incontrollable fashion. While the discovery of their genetic root causes has led to the development of effective drugs with limited side effects for specific types of cancer, most malignancies are still treated with chemotherapy, an indiscriminate approach that kills both cancerous and healthy cells, because we still don’t know the genetic abnormalities that drive their growth.

CRISPR is being used massively to add more pieces to the cancer puzzle by helping researchers identify unknown genetic mutations responsible for the growth of tumours, according to Dr Jonathan Schatz, Associate Professor at Sylvester Comprehensive Cancer Center of the University of Miami.

In a study published in Nature last April, researchers at Wellcome Sanger Institute reported that they had used CRISPR to tamper with thousands of genes from 30 cancer types to pinpoint those vital for cancer survival. The analysis led them to list about 600 genes that might offer promising targets for cancer drugs.

“CRISPR is an incredibly powerful tool that enables us to do science at a scale and with a precision that we couldn’t do five years ago,” Dr Kosuke Yusa, Sanger Institue faculty member in cellular genetics at the time and co-lead author of the research, said in a press release. “With CRISPR we have discovered a very exciting opportunity to develop new drugs targeting cancer.”

Not only does CRISPR boost the search for new cancer mutations, but it also provides better models of already known cancer abnormalities.

Dr Schatz’s team, for example, has used the gene editing technology to create the first lab mouse with a chromosomal abnormality associated with many blood cancers, in particular a type called anaplastic large cell lymphoma (ALCL) that is common in children and adolescents. Prior to the introduction of CRISPR, the best shot researchers had to replicate a tumour in mice was to modify their immune cells in such a way to force them to turn cancerous. This, however, was an imperfect model because it didn’t mimic the genetic root cause starting the proliferation of cancerous cells.

“CRISPR allowed us to build a model that’s more reflective of what’s happening in human patients with ALCL,” said Dr Schatz. “We can use this model to study the biology of the disease and create targeted treatments that hopefully will replace chemotherapy, which is still the main pharmacological approach for the disease.” The hope is to offer an effective therapy for the 40 percent of ALCL patients who can’t achieve a cure with current treatments.

Using the same gene editing strategy, the team is now turning to reproducing more mice models carrying mutations typical of blood cancers to speed up testing of new therapies.

With the power to both decode cancer genetic pathways and create ideal models for drug testing, CRISPR might pave the way for several new targeted treatments that fix the disease-causing alterations in the genes instead of simply killing tumour cells like old-school chemotherapy does.

 

 

This story was originally published in the 43th issue of Global Health Asia-Pacific magazine.

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