gene therapy

Mukherjee Mess-up, the Secret Harvard Meeting, and Success in Gene Therapy: May 2016 with Nathan and Laura

Today we look back on the genomics headlines over the past month (and a few days). To do this we’re joined by our regular commentators, Nathan Pearson and Laura Hercher.

First we take on the science journalism kerfuffle of the year. When Pulitzer Prize winning author, Siddhartha Mukerjee, got epigenetics wrong in his New Yorker piece, scientists came out en masse to denounce it. Nathan reassures us that scientists aren’t afraid of writers.

Then on to that secret meeting at Harvard, HGP-Write. Laura gives it two thumbs down, saying it’s very normal for folks to be scared of the idea of synthesizing a human genome from scratch. So don’t make it more scary with a secret meeting and total lack of transparency.

Finally, we review some positive success stories for gene editing, specifically some gene therapies which have been approved or undergoing new trials.

I Prefer My Bacon Crispy: Why I Don’t Think CRISPR is Really That Big of a Deal

With all the recent news around CRISPR my reaction is “meh” (coincidentally, the same reaction I’m having to the current US presidential election noise). We are a wee bit early for both.

Is Jurassic Park – and now Jurassic World real? No.

Zinc Finger Nucleases (ZFNs) with Sigma's Dave Smoller, Supriya Shivakumar

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Dr. Dave Smoller, CSO, Sigma Life Science Bio and Contact Info

Dr. Supriya Shivakumar, Global Marketing Manager, Sigma Life Science Bio and Contact Info

ZFN technology and products Listen (9:42) ZFN technology and products

A game changer Listen (2:09) "A game changer"

Rats better than mice for studying neural diseases Listen (3:42) Rats better than mice for studying neural diseases

Improvement in products Listen (2:12) Improvement in products

Why buy when researchers can make their own? Listen (3:00) Why buy when researchers can make their own?

Revival of gene therapy Listen (6:24) Revival of gene therapy?

Recent study on hemophilia Listen (5:59) Recent study on hemophilia

Applications in AgBio Listen (7:35) Applications in AgBio

If we'd only had ZFN back when Listen (7:55) If we'd only had ZFNs back when . . .

(ZFNs) or Zinc finger nucleases are a class of engineered DNA-binding proteins that enable targeted editing of the genome. They do this by creating double-strand breaks in DNA at virtually any specific location. Through homologous and non-homologous recombination, the technology can generate precisely targeted genomic edits including gene deletions (Knockouts), integrations, or modifications. The technology holds great promise for treating human disease such as hemophilia and sickle cell anemia and may revive gene therapy. In addition the ZFNs are offering new possibilities in plant and animal research. Developed first by Sangamo Biosciences, the technology has been licensed to Sigma Life Science which they offer as their CompoZr ZFN technology.

Here to talk to us about this exciting technology and the promise of its many applications is Dr. Dave Smoller, CSO at Sigma and Supriya Shivakumar, global marketing manager for functional Genomics at Sigma. Dave was formerly president of the Research Biotech Business Unit at Sigma. Before that he founded two companies which were acquired, ProteoPlex, focused on functional genomics and Genome Systems, which offered access to Human Genome project related technologies. Supriya did graduate studies in Harold Varmus’ lab at UC San Francisco, where she used reverse genetics approaches to study oncogenes and their normal biological role in C. elegans.

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