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Magical Molecular Scissors

Magical Molecular Scissors

15 Jul 14:00 by Lorraine Gowar

CRISPR and CRISPR Cas 9 in genome editing

The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and the CRISPR associated Cas system is the latest addition to genome editing derived from components of the bacterial immune system. CRISPR permits targeted gene editing offering a simple, rapid and broad application to genome editing.

In 2012 researchers had realized that CRISPR could be adapted, engineering the genomes of microbes and other cells. The adoption of CRISPR continues to be utilized in labs across the globe and its simplicity has revolutionized genome engineering.

How has CRISPR affected the world so far? The remarkable developments in CRISPR research affects gene therapy worldwide at present, I expect its progress and growth to be spectacular.

The CRISPR (Cas) system mainly consists of two major components: the Cas nuclease (Cas9) and the specific guide RNA (gRNA). They form a stable ribonucleoprotein (RNP) complex. The gRNA carrying Cas9 finds a specific DNA region and when this is matched the target DNA can be cleaved into a double stranded break. The Cas9 RNP complex can then be introduced into cells directly and act immediately without transcription or translation thus repairing the damage or malformation with relative ease.

Remarkable, yes! Ground-breaking, unquestionably!!!!!! The future of this revolutionary advance is exciting to say the least. So far CRISPR has created mosquitoes that don’t transmit malaria (fantastic!!) unusually muscular beagles (a little odd!!) and micro pigs (who knew!!!!) In addition to these interesting genomic adaptions that have varying effects on the world the evolution of health care is key.

My interest lies predominantly with this progression. It has been announced that $40 million dollars in funding has been awarded for, series A: human trials in Boston for people suffering from Duchenne Muscular Dystrophy which is a fatal condition. It is hoped trials will be commencing this year to work on a longer lasting solution for the sufferers who are primarily boys that loose the ability to walk in their teens and often die from complications such as heart or lung failure.

The funding in to this trial will be using CRISPR Cas9 to send a genetic instruction to the body to produce the protein dystrophin, a muscle protecting protein that Duchenne patients suffer from a lack of. Currently there are two drugs on the market to treat Duchenne that both slow the progression of the disease. Hopefully these trials will revolutionize treatment for the sufferers of Duchenne Muscular Dystrophy making their future a much brighter one.

The liver however, BEST organ ever!! Makes it easy to transport CRISPR throughout the body. Anything injected into your blood supply is very likely to reach the liver. CRISPR researchers are working on delivering fatty particles with CRISPR on board to components in the liver thus aiming to treat transthyretin amyloidosis.

Transthyretin amyloidosis is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Amyloid deposits in the nerves of the peripheral nervous system so can result in a loss of sensation in the extremities which is called peripheral neuropathy. This is horribly debilitating and painful and tends to result in the need for an extreme amount of health care requirements which can be the case for many diseases such as hepatitis.

Hepatitis B has been the top of the list for a researcher in the US who is using CRISPR to destroy this viral DNA that lingers in the liver. Hepatitis B is an inflammation of the liver through a virus known as HBV. Although a vaccine does exist, no cure does. Luckily, most people can be diagnosed early and successfully treated. Although If the infection becomes chronic, it is important to prevent and minimize liver damage. However CRISPR is hoping to cure those sufferers that miss the warning signs and as a result become chronically ill.

These conditions are awful for the patients and mostly result in the requirement for painful, expensive treatments. The development of CRISPR is expected to result in less expensive treatments and possible eradication of some diseases making the future extremely exciting!!

The big challenge is delivering the CRISPR machinery to tissues inside the body with precision, the research advances in this area are incredible and show no sign of slowing.

The future is CRISPRy.............