July 12, 2020

The Niche

Knoepfler lab stem cell blog

mitochondrial transfer

8 min read

The topic of heritable human genetic modification has been heating up recently. Prominent scientists, ethicists, and legal scholars have being weighing in, and there is a range of attitudes. Some favor a complete, moratorium including even lab work, while on the other end of the spectrum there are those who have a more liberal perspective. Many of us fall in the middle somewhere. I have been interested in having conversations with people with diverse views and posting them on this blog. You can see past …Read More

6 min read

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 of the mutations causing these diseases are in nuclear DNA, while others are in the mitochondrial DNA (mtDNA). The main current approach to prevention is preimplantation genetic diagnosis (PGD) …Read More

4 min read

Each year towards the end of December I make predictions for the coming year as I did for 2015. In the past I usually make a top 10 prediction list, but for this year I made 20 predictions. Admittedly some of them may have been more hopes than predictions. At mid-year today on June 30th, how am I doing with 2015 predictions? See below. Note that of course for some the jury is still out. BTW, stay tuned for more on an upcoming update …Read More

5 min read

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) to reduce the burden of mutant mitochondrial DNA (mtDNA). Their work was done primarily in mice, but also using chimeras made with murine oocytes fused with human cells bearing …Read More