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Analysis: human immunity to Cas9 bigger #CRISPR therapeutic hurdle than off-targets?

April 6, 2018 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Some in CRISPR-Cas9-land who are focused on potential future clinical applications are kind of rejoicing or at least sighing a breath of relief. This upbeat swing in the atmosphere (from investors especially) was sparked by retraction of that paper, the one initially reporting tons of supposed off-target CRISPR-Cas9 activity in mice, which turned out to be a “nothing […]

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Journal club: Jaenisch lab paper on epigenetic CRISPR-Cas9 rescue of Fragile X in a dish

February 26, 2018 / Professor Paul Knoepfler, Ph.D. / 3 Comments

Liu-et-al.-Figure-6C-Fragile-X-CRISPR-Cell-e1519673510685

There’s much more to CRISPR-Cas9 than just gene editing and a new paper from the lab of Rudy Jaenisch in Cell highlights that in an exciting way. It reports epigenetic reversal of a Fragile X Syndrome phenotype in induced pluripotent stem cell (IPSC) neurons. Fragile X Syndrome is a neurological disorder in boys resulting from CGG repeat

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Keep calm & CRISPR on: perspectives on report of human Cas9 immunity

January 9, 2018 / Professor Paul Knoepfler, Ph.D. / 7 Comments

The news that CRISPR-Cas9 gene editing in its current form may not work in a substantial fraction of people due to many of us having immunity to Cas9 came as a shock to many, but if you think about it, maybe it’s not so surprising. I don’t see it as the end of the world. A (preprint) from

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CRISPR critters: Cute pics of Cas9 gene edited animals

May 24, 2015 / Professor Paul Knoepfler, Ph.D. / 2 Comments

As CRISPR gene editing technology has advanced in the last few years, the number of genetically modified animals made with this system has steadily increased. Some are very interesting and useful for science. At the same time especially when they are little, they can be very cute. A nickname is going around for these GM

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Doudna most deserving of CRISPR Cas9 patent, says poll

May 3, 2015 / Professor Paul Knoepfler, Ph.D. / 4 Comments

With more than 250 votes cast, Jennifer Doudna is leading by a decisive margin in a poll asking who deserves the patent for use of CRISPR-Cas9 as a tool. Feng Zhang got only just over half the votes of Doudna. Another take home message from this poll is that a sizable minority–almost one in five– said

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Who deserves patent for CRISPR-Cas9 in human cells? Take our poll

April 21, 2015 / Professor Paul Knoepfler, Ph.D. / 6 Comments

There is no hotter technology than CRISPR-Cas9 gene editing tools. Perhaps it is not surprising then that there is a patent dispute over it, which falls into two camps: (1) Jennifer Doudna & Emmanuelle Charpentier, and (2) Feng Zhang. Who deserves the intellectual property for use of CRISPR-Cas9 in human cells? Take our poll.

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5 new cool papers on CRISPR Cas9 gene editing

April 8, 2015 / Professor Paul Knoepfler, Ph.D.

CRISPR Cas9 is a relatively new, powerful approach to gene editing and genetic modification more generally. In the relatively short window of time in which this technology has been widely available for gene editing applications it has already evolved rapidly to become more powerful. Here are some new papers of interest on this technology. With

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CRISPR Cas9 Gene Editing Tech Will Revolutionize Biology

March 12, 2014 / Professor Paul Knoepfler, Ph.D. / 8 Comments

Why is CRISPR Cas9 gene editing such a big deal? Imagine being able to make mutations in any gene you wanted say in stem cells in a relatively simple, quick and affordable fashion? Sounds exciting, huh? Every so often a new kind of technology comes along in the biomedical sciences that is a true game

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