CRISPR-CAS9

Clustered regularly-interspaced short palindromic repeats

HOW IT WORKS

CRISPR, truly just about three years old, allows researchers to potentially change the DNA of any organism – including humans. It’s an inexpensive, fast-working technology that has researchers envisioning new ways to eliminate disease, create hardier plants, wipe out pathogens and more

CRISPR

$20-$40

per gene

200-times less expensive

CRISPR can achieve complete protein loss as compared to only partial protein reduction via RNAi.

TALEN

$4000

per gene

Months longer to perform than using CRISPR

RNA interference research has historically been plagued by off-target effects that complicate the interpretation of experiments.

COST

TIMELINE

Key events in the CRISPR story

July 1995

CSRISPR sequences are found to be common in other microbes.

A cluster of biotech companies has sprung up to use CRISPR technology

October 2011

CARIBOU BIOSCIENCES

Berkeley, California

Focus: Research, industry, therapeutics, agriculture

Raised: $11 Million

March 2013

The University of California and others file for a patent on findings.

November 2014

INTELLIA THERAPEUTICS

Cambridge, MA

Focus: Therapeutics

Raised: $15 Million

April 2015

Researchers report that they have edited human embryos with CRISPR, triggering an ethical debate.

December 1987

Researchers find CRISPR sequences in Escherichia coli, but do not characterize their function.

March 2007

Scientists at food company Danisco determine that the repeats are part of a bacterial defense against viruses.

June 2012

Researchers report that CRISPR can be used to perform genome editing.

January 2013

CRISPR is used in mouse and human cells, fuelling rapid uptake of the technique by researchers.

November 2013

EDITAS MEDICINE

Cambridge, Massachusetts

Focus: Therapeutics

Raised:  $43 Million

April 2014

MIT and the Broad Institute are granted a patent on CRISPR gene editing, sparking a fierce patent battle.

March 2015

Report of the first CRISPR gene drive, which can spread an edited gene rapidly through a population.

November 2013

CRISPR THERAPEUTICS

Basel, Switzerland

Focus: Therapeutics

Raised:  $89 Million

FUNDING & PATENTS

Published Patent Applications

In 2014, worldwide patent applications that mention CRISPR leapt and a patent battle intensified.

62

57

34

29

28

Massachusetts Institute of Technology (MIT)

Broad Institute

MIT bioengineer Feng Zhang

Dansico

Dow Agrosciences

 

Funding

 

A sharp jump in US National Institutes of Health funding for projects involving CRISPR is a harbinger of future advances.

BIRTH INJURY APPLICATIONS

Stem cell therapy research has had mixed results in clinical trials, but such therapies offer hope to families with children who have disabilities, who believe that the procedure can help their children’s symptoms.

Much research into the mechanisms of birth injury has gone to the study of how to help children with birth injuries using stem cell therapies – a field in medicine that is still in its infancy but has been garnering a great deal of attention in the media recently, especially with the rising trend of cord blood banking.

SPEED AND LOW COST OF RESEARCH

TRAUMATIC BRAIN INJURIES

DISABILITIES

In some cases, stem cell therapy has helped adults with traumatic brain injuries regain motor and/or cognitive function, and clinical trials are currently underway. However, the brain structure and function of adults and newborns can differ significantly, so this is a development that must be approached with cautious optimism.

Much of the media attention surrounding this gene editing technique focuses on the speed and low cost of research. The technique is, in the end, a tool – it increases the speed of research and allows researchers to successfully conduct gene editing, an important part of stem cell therapies.

Disabilities such as hypoxic ischemic encephalopathy (HIE) and cerebral palsy are not generally genetic. Gene therapies, however, have, in some cases, seemed to improve functional outcomes in cerebral palsy and HIE, though more rigorous testing is needed to fully understanding when, how, and to what extent gene therapies are effective.

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