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Weekly reads: ‘junk DNA’, COVID chaos, CRISPR CasΦ, & postdoc opps, more

July 19, 2020 February 1, 2021 / By Professor Paul Knoepfler, Ph.D. / 2 Comments

Here are our weekly recommended regenerative medicine and other notable science reads including a few things on COVID. It’s been quite an interesting week. “Junk DNA” is not so junky, role in differentiation NIH scientists showed how ancient retroviral genes, or “junk DNA”, may play a role in helping stem cells decide to become neurons. This …

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The Niche COVID-19 updates & resources

stem-cells-for-COVID-19-infographic_small

The novel coronavirus and the disease it causes, COVID-19, have swept the world with few proven treatment options. Vaccines are only recently being rolled out the last 2 or so months, which gives some real hope. While the novel coronavirus-caused illness is generally a mild disease in kids (many of whom don’t even show symptoms), …

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Right To Try law has a big loophole that’s an opening for bad actors

October 7, 2019 September 28, 2022 / By Professor Paul Knoepfler, Ph.D. / 14 Comments

There appears to be a big, risky loophole in the relatively new national Right To Try law. Some folks apparently anticipated this problem long ago, but I think most of us weren’t aware of it. Update: More specifically, some law and policy experts were writing about this and other potential loopholes in 2018. I recommend …

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Richard Garr Q&A on his new Right-To-Try firm Beacon of Hope

September 12, 2019 February 1, 2021 / By Professor Paul Knoepfler, Ph.D. / 6 Comments

A new right-to-try company called Beacon of Hope is stirring some intense discussion. State and federal right-to-try laws could potentially change the fabric of how many investigational studies are conducted. That may happen through firms like Beacon of Hope. However, we don’t know much about the firm. I’m hoping to help change that. Today’s post …

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Counterpoints to Lovell-Badge & Daley’s CRISPR baby rationales

February 11, 2019 February 1, 2021 / By Professor Paul Knoepfler, Ph.D. / 5 Comments

Two prominent scientists, Robin Lovell-Badge and George Daley, have been amongst the most outspoken proponents of leaving the door open to heritable human genetic modification via CRISPR. While they each have articulated their reasons in somewhat different ways at times, their core reasons arguing in favor of future heritable CRISPR appear largely the same. In …

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Trying to connect the dots on CRISPR baby story paints a dark, cloudy picture

November 27, 2018 February 1, 2021 / By Professor Paul Knoepfler, Ph.D. / Leave a Comment

When I was a little kid I played this game called “connect the dots” or “dot-to-dot”, where you draw a line from dot-to-dot in numerical order and at some point a picture starts to emerge. I think kids and even some adults still play this today. They even come in extreme versions with hundreds or …

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Could Cancer Risk Claw CRISPR’s Potential? Some Balanced Perspectives

June 13, 2018 February 1, 2021 / By Professor Paul Knoepfler, Ph.D. / 2 Comments

Could potential associated cancer risks claw into CRISPR’s potential? The short answer from both previous and new data is that while CRISPR gene editing impacts the P53 pathway, which is involved in cancer along with having many other functions, this news is neither too surprising nor a fatal flaw, but some caution is warranted. CRISPR …

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Updated FDA granted RMAT List (85)

Today’s post includes a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT. Sometimes people call these “r mat” with a space. As of December 12, 2023 there are 85 RMATs in the public domain. I also include FDA data on RMAT submissions via an FDA RMAT resource …

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Update on CIRM’s future: will it include 2020 measure?

December 3, 2017 February 1, 2021 / By Professor Paul Knoepfler, Ph.D. / 1 Comment

The California Institute for Regenerative Medicine, more widely known as CIRM, has accomplished big things over the course of its history of about a decade and sparked a great deal of innovation, but what does the future hold for our stem cell agency? CIRM has a new, respected President and CEO Maria Millan solving one …

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7 tech hurdles to human germline CRISPR

November 6, 2017 February 1, 2021 / By Professor Paul Knoepfler, Ph.D.

Human germline CRISPR raises major bioethical considerations, but what about technical issues? Setting aside the many ethical issue about the general idea of human modification itself, could this really work? Yes in theory it could, but there are some very tough technological challenges that could and likely would cause failures or unacceptable outcomes at many steps …

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