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CRISPR @MIT

Curator & Reporter: Aviva Lev-Ari, PhD, RN

FIRST, there was Jennifer A. Doudna @ Berkeley

Along with colleagues at UC Berkeley and in Sweden, Doudna, in 2012, discovered a gene-editing technique called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). The technology “gives researchers the equivalent of a molecular surgery kit for routinely disabling, activating or changing genes,” wrote Science magazine in a Dec. 2013 article naming CRISPR one of its runners-up for breakthrough of the year. CRISPR “has become red hot in the past year,” the journal article said.

http://www.fnih.org/press/releases/foundation-nih-award-lurie-prize-biomedical-sciences-jennifer-doudna-uc-berkeley

New DNA-editing technology spawns bold UC initiative

By Robert Sanders, Media Relations | March 18, 2014

BERKELEY —The University of California, Berkeley, and UC San Francisco are launching the Innovative Genomics Initiative (IGI) to lead a revolution in genetic engineering based on a new technology already generating novel strategies for gene therapy and the genetic study of disease.The Li Ka Shing Foundation has provided a $10 million gift to support the initiative, establishing the Li Ka Shing Center for Genomic Engineering and an affiliated faculty chair at UC Berkeley. The two universities also will provide $2 million in start-up funds.At the core of the initiative is a revolutionary technology discovered two years ago at UC Berkeley by Jennifer A. Doudna, executive director of the initiative and the new faculty chair. The technology, precision “DNA scissors” referred to as CRISPR/Cas9, has exploded in popularity since it was first published in June 2012 and is at the heart of at least three start-ups and several heavily-attended international meetings. Scientists have referred to it as the “holy grail” of genetic engineering and a “jaw-dropping” breakthrough in the fight against genetic disease. In honor of her discovery and earlier work on RNA, Doudna received last month the Lurie Prize of the Foundation for the National Institutes of Health.

 

“Professor Doudna’s breakthrough discovery in genomic editing is leading us into a new era of possibilities that we could have never before imagined,” said Li Ka-shing, chairman of the Li Ka Shing Foundation. “It is a great privilege for my foundation to engage with two world-class public institutions to launch the Innovative Genomics Initiative in this quest for the holy grail to fight genetic diseases.”

http://newscenter.berkeley.edu/2014/03/18/new-dna-editing-technology-spawns-bold-uc-initiative/

Ribozymes and RNA Machines – Work of Jennifer A. Doudna

http://pharmaceuticalintelligence.com/2013/04/15/ribozymes-and-rna-machines-work-of-jennifer-a-doudna/

Gene Therapy and the Genetic Study of Disease: @Berkeley and @UCSF – New DNA-editing technology spawns bold UC initiative as Crispr Goes Global

http://pharmaceuticalintelligence.com/2014/03/27/gene-therapy-and-the-genetic-study-of-disease-berkeley-and-ucsf-new-dna-editing-technology-spawns-bold-uc-initiative-as-crispr-goes-global/

Evaluate your Cas9 Gene Editing Vectors: CRISPR/Cas Mediated Genome Engineering – Is your CRISPR gRNA optimized for your cell lines?

http://pharmaceuticalintelligence.com/2014/03/25/evaluate-your-cas9-gene-editing-vectors-crisprcas-mediated-genome-engineering-is-your-crispr-grna-optimized-for-your-cell-lines/

CRISPR-Cas: A powerful new tool for precise genetic engineering

http://pharmaceuticalintelligence.com/2013/11/29/crispr-cas-a-powerful-new-tool-for-precise-genetic-engineering/

THEN, there was development of CRISPR Applications @MIT using a precise way to delete and edit specific bits of DNA—even by changing a single base pair. This means they can rewrite the human genome at will.

Genome Surgery

• by Susan Young

Over the last decade, as DNA-sequencing technology has grown ever faster and cheaper, our understanding of the human genome has increased accordingly. Yet scientists have until recently remained largely ham-fisted when they’ve tried to directly modify genes in a living cell. Take sickle-cell anemia, for example. A debilitating and often deadly disease, it is caused by a mutation in just one of a patient’s three billion DNA base pairs. Even though this genetic error is simple and well studied, researchers are helpless to correct it and halt its devastating effects.

Now there is hope in the form of new genome-engineering tools, particularly one called CRISPR. This technology could allow researchers to perform microsurgery on genes, precisely and easily changing a DNA sequence at exact locations on a chromosome. Along with a technique called TALENs, invented several years ago, and a slightly older predecessor based on molecules called zinc finger nucleases, CRISPR could make gene therapies more broadly applicable, providing remedies for simple genetic disorders like sickle-cell anemia and eventually even leading to cures for more complex diseases involving multiple genes. Most conventional gene therapies crudely place new genetic material at a random location in the cell and can only add a gene. In contrast, CRISPR and the other new tools also give scientists a precise way to delete and edit specific bits of DNA—even by changing a single base pair. This means they can rewrite the human genome at will.

original article in Technology Review

http://www.technologyreview.com/review/524451/genome-surgery/

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