January 23, 2021

The Niche

Trusted stem cell blog & resources

Parkinson’s Disease

4 min read

I’m playing catch-up on some reading given how busy I’ve been and this includes a groundbreaking NEJM pub on CRISPR for Sickle Cell and Thalassemia. CRISPR for Sickle Cell From December, here’s the key paper in the NEJM: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. There’s a lot to like about this clinical trial paper and it gives real hope to patients with Sickle-Cell Disease and Thalassemia. It takes an interesting, indirect clinic approach to addressing Sickle Cell by turning off a …Read More

3 min read

I find following both stem cell news and the stream of interesting publications to be kind of fun and thought-provoking. This past week or two has been very busy. Here is a news summary and list of notable pubs. Stem Cell News on Parkinson’s Disease There were a number of interesting developments on the Parkinson’s Disease front. Over at STAT News, Sharon Begley wrote about a “secret” experiment in this piece: A secret experiment revealed: In a medical first, doctors treat Parkinson’s with a …Read More

2 min read

Where do things stand today in 2020 with IPS cell research? It’s been 14 years since they were first reported, but they continue to make news. Back in 2006 I was wrapping up my postdoc with Bob Eisenman at The Hutch in Seattle, largely studying Myc, when Shinya Yamanaka published his first induced pluripotent stem cell (IPS cell) paper in Cell. For me it was one of those amazing moments reading a pub when you just go “wow!” Now all these years later we …Read More

2 min read

By Jeanne Loring 10, 9, 8, 7, 6, 5, 4, 3, 2, 1 blast off! It’s good to get out of the lab occasionally to get a different perspective.   Last Thursday, December 5, I got a very different perspective, in Cape Canaveral, Florida, where I got to watch the SpaceX Dragon spacecraft be launched by a Falcon 9 rocket. I was there because two years ago my research team was offered the opportunity to design an experiment for the International Space Station (ISS).  The …Read More

2 min read

Japan is a leader in stem cell and regenerative medicine research, and in particular in clinical translation toward the bedside. Induced pluripotent stem cell (IPSC)-based investigational therapies are rightly an area of focus in Japan given their invention by Nobel Laureate Shinya Yamanaka. Small clinical studies based on IPSC have been initiated there for several diseases including related to vision loss and Parkinson’s Disease. David Cyranoski over at Nature reports on a new, different stem cell clinical effort in Japan for vision loss with …Read More

3 min read

In Parkinson’s Disease patients develop neurological dysfunction as they lose a special kind of brain cell called dopaminergic (or dopamine) neurons. While a number of different approaches to this disease have been studied for decades, nothing has proven particularly successful in slowing its progression. As a result there has been a big need for novel thinking about how to tackle Parkinson’s Disease including via stem cells. One of the most exciting ideas has been to use human induced pluripotent stem cells (hIPSC) or human embryonic …Read More

2 min read

One of the highlights of Day 1 of #ISSCR2018 for me so far was the talk by Lorenz Studer (Co-Founder of BlueRock) on the use of human embryonic stem cell (HESC)-derived dopamine neurons for Parkinson’s Disease. Note that for this post and if I have time any others on this meeting, they are probably going to be somewhat stream of consciousness notes from the talks and may have some outlined points rather than sentences. I have some questions or notes for myself that I …Read More

2 min read

One of the most famous living biological scientists, Sir Ian Wilmut, just announced that he has Parkinson’s Disease. I wish him the best in dealing with this illness. Wilmut is very well-known for having cloned the first mammal, Dolly the Sheep. This work followed on the earlier breakthrough by Sir John Gurdon of cloning the first vertebrate with his work in frogs. it just occurred to me: I’m not sure if the first cloned frog had a name! Over the years there has been …Read More

4 min read

By Jeanne Loring There are ten million people in the world who have Parkinson’s disease. 125,000 of these are living in California.  People with the disease often have to step away from their jobs because the main symptoms – tremor or freezing up of muscles – make it difficult to get through a whole day of work. Parkinson’s disease is caused by the loss of a specific neuron type in the brain.  The dopamine neurons in the region of the brain called the substantia …Read More

4 min read

New human clinical trials using derivatives of pluripotent stem cells in China for Parkinson’s Disease (PD) have raised expectations and some eyebrows. PD is a neurodegenerative condition, sometimes diagnosed or followed by PET scans such as the one at left, characterized by loss of dopaminergic neurons leading to severe and sometimes life-threatening symptoms. Pluripotent stem cells are powerful as their name implies and they have great clinical potential, but if they are not utilized properly they have robust tumor forming potential. This risk can …Read More