Let’s start with some stem cell good news on evolving approaches to treat sickle cell disease involving stem cells. Then on another related front things were more concerning.
Stem cell good news on sickle cell disease, a complication
Sickle Cell Disease News reports Adding Briquilimab Boosts Success of Sickle Cell Stem Cell Transplant. This is a story some of enhancing outcomes. It’s an exciting time in this regenerative area more generally as gene editing approaches to sickle cell also look very encouraging.
On the flip side, Endpoints has some discouraging news on gene editing for sickle cell: Sickle cell gene editing startup halts trial after flagging serious side effect in first patient. This is about Graphite Bio’s CEDAR trial of nula-cel. A trial participant developed pancytopenia. It’s unclear how the experimental approach might have caused this adverse event. Here’s the nula-cel trial listing. Other gene editing approaches to sickle cell seem more encouraging.
More recommended reads
Transplanting rejuvenated blood stem cells extends lifespan of aged immunocompromised mice, npj Regenerative Medicine. Can this potentially be done with people?
Integrated intracellular organization and its variations in human iPS cells, Nature. This paper feels like a resource with more than 200,000 images of cells at the resolution of organelles, which allows drawing some conclusions about variation in iPS cells.
1 thought on “Weekly reads: stem cell good news, bad news on sickle cell, MS, IPSCs”
In reference to your video on T1D. Vertex bought ViaCyte last year. Vertex has began using their islets-derived from stem cells for transplants into liver of T1D patients using immunosuppressive therapy to prevent rejection.