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Mitalipov Nature pub review: IPSC & SCNT for mitochondrial disease

July 15, 2015 / Professor Paul Knoepfler, Ph.D. / 6 Comments

In a new, thought-provoking paper today in Nature, Shoukhrat Mitalipov and a multi-institutional team report a significant advance toward potential novel ways to treat mitochondrial diseases. What are these illnesses? Mitochondrial diseases are rare, but devastating disorders caused by genetic mutations. Today they are largely impossible to treat in meaningful ways other than palliative care. Some […]

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Juan Carlos Izpisua Belmonte Cell Paper on Gene Editing for Mitochondrial Disease

April 23, 2015 / Professor Paul Knoepfler, Ph.D. / 2 Comments

The Juan Carlos Izpisua Belmonte group published a Cell paper today on using gene editing to reverse mutations associated with human mitochondrial disease. The paper is Reddy, et al. and is entitled, “Selective Elimination of Mitochondrial Mutations in the Germline by Genome Editing”. The authors report success using TALEN-based gene editing or mitochondrial-direct restriction enzyme (mito-ApaLI)

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My concerns about Nature paper on Genome Transfer for mitochondrial disease

December 20, 2012 / Professor Paul Knoepfler, Ph.D. / 30 Comments

A paper just came out in Nature yesterday entitled: “Nuclear genome transfer in human oocytes eliminates mitochondrial DNA variants.” It’s surely risky of me to say this, but is this paper really a clinically-relevant breakthrough for mitochondrial disease worthy of a Nature paper? The study as science is fine and very interesting, but it seems quite

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Perspectives on uncritical NYT coverage of cardiac mitochondrial transplant trial

July 11, 2018 / Professor Paul Knoepfler, Ph.D. / 12 Comments

One of the reasons I’m a scientist is that I find biology fascinating and admittedly novel biomedical science can really catch my eye, but more recently as a somewhat grizzled researcher, I’ve become increasingly skeptical about some “sexy” research and media coverage of it. A small red flag went up for me as I was

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Mitochondrial Replacement Hype Goes Nuclear Including by Wellcome Trust

June 9, 2016 / Professor Paul Knoepfler, Ph.D. / 4 Comments

A new paper from a UK team yesterday showed that mitochondrial replacement therapy faces a serious and difficult safety hurdle fitting with similar findings from an earlier recent study from a different team led by Dieter Egli, but the UK media and some scientists there are engaging in hype, claiming the exact opposite. They are pushing the unscientific

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New Herbert lab Nature paper reinforces mitochondrial replacement Achilles heel

June 8, 2016 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Today a new Nature paper from Dr. Mary Herbert’s group in the UK has found a key problem with mitochondrial replacement therapy that fits with data from others. Mitochondrial replacement data The goal of preventing mitochondrial disease using various kinds of genome transfer technologies is a noble one, but mitochondrial replacement therapy has faced a

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UK should freeze mitochondrial replacement as Egli paper ID’s serious problem

May 19, 2016 / Professor Paul Knoepfler, Ph.D. / 1 Comment

An important new Cell Stem Cell paper from Dieter Egli’s lab points to an inherent, serious problem with so-called 3-person IVF or mitochondrial replacement technology that warrants putting an immediate hold on all efforts to use it in humans. I have pasted the graphical abstract from the paper below. For years a few of us scientists and

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250,000-fold oversight on 3-person IVF mitochondrial transfer?

June 23, 2015 / Professor Paul Knoepfler, Ph.D. / 4 Comments

Remember the debate over so-called 3-person IVF? The goal of this technology, also referred to as mitochondrial transfer and 3-parent IVF, is to prevent mitochondrial disease through nuclear transfer in oocytes or one-cell embryos. The resulting genetically modified (GM) human embryos and ultimately children if it works could have dodged mitochondrial disease, but also could have serious or

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Monday, March 2: My Reddit AMA on 3-Person IVF, Mitochondrial Transfer

February 27, 2015 / Professor Paul Knoepfler, Ph.D.

On Monday, I’ll be doing another Reddit AMA, this time on 3-person IVF also known as 3-parent IVF, mitochondrial transfer, 3-parent babies, etc. You can see the upcoming AMAs including mine listed in the right sidebar here. Mine will start at 10AM PST/1PM EST. I hope to discuss with a wide audience all the key

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Myths about 3-person IVF mitochondrial transfer

February 10, 2015 / Professor Paul Knoepfler, Ph.D. / 11 Comments

It’s been somewhat of a helter-skelter time for the new technology often referred to as 3-person IVF or mitochondrial transfer as the UK considers whether to legalize this experimental technology for use in humans. I believe that this technology is not ready now for use in humans and for more background on why as well

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