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Targeting killer diseases under the microscope

It is a depressing statistic, but every single minute, a child somewhere in the world dies from malaria. The World Health Organization (WHO) estimates that, globally, 3.4 billion people are at risk of the tropical disease which, according to the WHO, caused an estimated 627,000 deaths in 2012.

Nonetheless, science is continuing its fightback. At GlaxoSmithKline (GSK), the British pharmaceutical giant, scientists and researchers have been working in conjunction with the Path Malaria Vaccine Initiative (MVI) on a vaccine – known as RTS,S – which could transform the battle against malaria.

"Making a vaccine against a parasite is a world first, because they are very, very complex," Dr Moncef Slaoui, Chairman of Global Research & Development and Vaccines at GSK, told Episode 6 of CNBC's Industrial Revolutions. "With one vaccine we take two stabs at this parasite: when it gets in the blood and when it hides in our liver for a period of days before it makes us sick."

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In late 2013, GSK released results of their large-scale Phase III trial of RTS,S. The trial showed that the vaccine reduced the number of malaria cases in young children by nearly half. In infants, the number of malaria cases fell by around a quarter.

"The work that has taken place over the past 18 months… has really been to understand the impact that these levels of protection can have on toddlers and infants in sub Saharan Africa," Slaoui told CNBC.com in a phone interview.

"Once we started to express them as the number of cases prevented for one thousand children immunized, our conclusion… [became] very clear: that this actually should be introduced and used," he added.

GSK hopes to have regulatory approval for the vaccine this year. It's been a long road – nearly 30 years – according to Slaoui, but worth it.

"I grew up in an environment where it was completely normal for a child to die," Slaoui said. "Being part of making it not normal for a child to die is great… while it's not perfect, it's already an incredible improvement in the lives of these people," he added.

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While researchers such as Slaoui are leading the field when it comes to vaccines, new discoveries in the field of genetics could radically alter the way we both prevent and treat debilitating, life threatening medical conditions.

At the University of California, Berkeley, Professor Jennifer Doudna and her team have been investigating the potential of the CRISPR-Cas9 system, which allows scientists to permanently modify genes.

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"People should be excited about Cas9 because it is a precision DNA 'scissors'," Doudna told Episode 6 of CNBC's Industrial Revolutions.

"It allows scientists to break DNA at sites where there might be a mutation, and allows cells to repair that mutation by introducing new genetic information," she added.

The potential of CRISPR is huge, with the scientific community exploring the possibility of treating everything from cancer to sickle cell anaemia and Huntington's disease using the technique.

The excitement surrounding CRISPR has increased in the last few years thanks to the publication of research co-authored by Doudna.

"The reason this came to many people's attention as a tool in genetics was in 2012 when I published a paper, together with a collaborator, Emmanuelle Charpentier, [on] the discovery that in one type of CRISPR system, there's a protein [Cas9] that is essentially a molecular 'scissors'," Doudna told CNBC.com in a phone interview.

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"We can programme it to cut different sequences of double stranded DNA… which turns out to be a very useful way to introduce new genetic information into cells," she added.

Earlier this year, the Broad Institute of MIT and Harvard announced that it had been awarded the first patent for "an engineered CRISPR-Cas9 system that is enabling scientists to modify genes and better understand the biology of living cells and organisms."

Feng Zhang, a Core Member of the Broad Institute and senior author of a 2013 paper in Science which showed that Cas9 could alter 'mammalian' cells' makeup, is a co-founder of Editas Medicine, a Massachusetts based start-up backed by $43 million of venture capital. Doudna is also one of Editas Medicine's founders.

Just how excited should we be about the work that Doudna has been contributing to? "Within 50 years' time, we will see the ability to repair many kinds of genetic disorders that are currently untreatable with current medicine," Doudna said.

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