29 January 2015

Gene therapy instead of pills?

Astra Zeneca to target drugs at genome sites

Pallab Ghosh, BBC Science Department

Pharmaceutical company Astra Zeneca has announced the launch of a research program aimed at developing a new generation of medicines designed to treat many diseases of a genetic nature.

The program is based on the newly created CRISPR methodology and will be implemented in collaboration with four leading academic and industrial research centers that specialize in the field of genomic research.

Dr. Lorenz Mayr, Vice President of Astra Zeneca, expressed hope that these studies will accelerate the development of new drugs for the treatment of diseases such as diabetes, atherosclerosis and many types of cancer.

The human genome contains about 24 thousand genes,
and defects in them can be the cause of many diseases

The decoding of the human genome 15 years ago raised the hope that doctors would be able sooner or later to determine which genetic disorders give rise to specific diseases, and then begin to develop drugs for their treatment.

At that time, great hopes were pinned on this. Speaking on American television immediately after the announcement of the first decoding of the human genome, then US President Bill Clinton said: "The children of our children will know about cancer only that this word means a constellation." He welcomed the completion of the project, which took 10 years and billions of dollars were spent.

However, these expectations have not been fulfilled – so far there are only a small number of drugs based on the results of decoding the human genome, and cancer remains a deadly disease.

When trying to understand the nature of the connection of gene defects with common diseases, scientists have encountered two main problems. Firstly, it was soon established that these pathologies are caused by many combinations of dozens of genes.

Secondly, methods for isolating specific genes are still extremely expensive and require a long time. Scientists have to develop their own "genetic scissors" for each specific gene, which allow them to cut it out of the DNA chain. This process can take several months.

However, over the past few years, methods have been developed for highly selective activation and inhibition of genes based on the CRISPR system – this is the English abbreviation of the term "short palindromic repeats regularly arranged in groups."

The method of site-selective genome editing with the help of an enzyme that recognizes the necessary sequence of the DNA chain on the tip of a complementary RNA "guide" promises revolutionary changes in the research and treatment of a number of diseases, from cancer and incurable viral diseases to hereditary genetic disorders such as sickle cell anemia and Down syndrome.

The new research program will involve the Wellcome Trust Sanger Institute, the universities of Berkeley and San Francisco, as well as the Broad/Whitehead institutes located in Massachusetts and the Thermo Fisher Scientific company.

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