September 24, 2020

The Niche

Knoepfler lab stem cell blog

mitochondrial disease

7 min read

It’s a particularly exciting time for the stem cell field. One of the most notable developments in the last year or so is the production and preliminary study of a totally new type of human embryonic stem cells (ESC) made by nuclear transfer instead of using leftover in vitro fertilization (IVF) embryos. This process of so-called therapeutic cloning has the power to produce patient specific ESCs called NT-ESCs that can in principle be used in the future for autologous transplants for a number of diseases …Read More

5 min read

Dear UK Parliament and Science and Technology Committee, I am writing to you about your deliberations on “mitochondrial donation” (also known as 3-parent technology) intended for the purpose of preventing heritable mitochondrial disorders. I am concerned about the Department of Health’s recent draft regulations that would allow 3-parent experiments to go forward and the possibility that the UK Parliament may vote to allow it. This experimental technology has a noble goal, but in my opinion there are too many unanswered questions and risks that remain to allow …Read More

3 min read

For the first time ever, scientists have successfully used somatic cell nuclear transfer (SCNT) via the process of therapeutic human cloning to generate normal human embryonic stem cells (hESC). Recall that there are two kinds of human cloning: therapeutic (which is reported in the new paper discussed in this post) and reproductive, which is making an actual new person with an identical genome to an existing person. The latter has never been achieved, but some of us are worried it is coming sooner than …Read More

2 min read

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 questionable to me to so strongly invoke clinical relevance as they do (even if they say some stuff must be discussed first): Before proceeding with human clinical trials on …Read More