May 30, 2020

The Niche

Knoepfler lab stem cell blog

Alejandro Ocampo

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

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

2 min read

Can hearts repair themselves via stem cell-like mechanisms? Sometimes what we scientists all know to be true turns out later on to be wrong and there are clear instances of this in the stem cell field. For example for decades the dogma was that the adult mammalian brain did not have stem cells, but now everyone knows that the adult brain does have stem cells. What we perceive as factual can change over time. Yamanaka disproved the entrenched notion that differentiated cells were permanently locked into …Read More