YOU CAN LISTEN TO THIS BLOG AS AN AUDIOCAST ON SPOTIFY Developing new tools to edit genes Having the right tools to do a job is important. Try using a large screwdriver to tighten the screws on your glasses and you quickly appreciate that it’s not just the type of tool that’s important, it’s also … Continue reading Tiny tools for the smallest of tasks, editing genes
Features
CIRM funds clinical trials targeting heart disease, stroke and childhood brain tumors
Gary Steinberg (Jonathan Sprague) Heart disease and stroke are two of the leading causes of death and disability and for people who have experienced either their treatment options are very limited. Current therapies focus on dealing with the immediate impact of the attack, but there is nothing to deal with the longer-term impact. The CIRM … Continue reading CIRM funds clinical trials targeting heart disease, stroke and childhood brain tumors
Gene therapy is life-changing for children with a life-threatening brain disorder
If you have never heard of AADC deficiency count yourself lucky. It’s a rare, incurable condition that affects only around 135 children worldwide but it’s impact on those children and their families is devastating. The children can’t speak, can’t feed themselves or hold up their head, they have severe mood swings and often suffer from … Continue reading Gene therapy is life-changing for children with a life-threatening brain disorder
CIRM funded trial may pave way for gene therapy to treat different diseases
Image Description: Jordan Janz (left) and Dr. Stephanie Cherqui (right) According to the National Organization for Rare Disorders (NORD), a disease is consider rare if it affects fewer than 200,000 people. If you combine the over 7,000 known rare diseases, about 30 million people in the U.S. are affected by one of these conditions. A … Continue reading CIRM funded trial may pave way for gene therapy to treat different diseases
Saying thanks and farewell to a friend
Tom Howing In this job you get to meet a lot of remarkable people, none more so than the patients who volunteer to take part in what are giant experiments. They are courageous pioneers, willing to be among the first people to ever try a new therapy, knowing that it may not help them and, … Continue reading Saying thanks and farewell to a friend
Stem cell gene therapy for Fabry disease shows positive results in patients
Darren Bidulka rests after his modified blood stem cells were transplanted into him at the Foothills Medical Centre in Calgary in 2017, allowing him to stop his enzyme therapy. (From left): Dr. Jeffrey Medin, Medical College of Wisconsin, Dr. Aneal Khan, the experimental trial lead in Calgary, and Darren Bidulka. Image Credit: Darren Bidulka Fabry … Continue reading Stem cell gene therapy for Fabry disease shows positive results in patients
Everything you wanted to know about COVID vaccines but never got a chance to ask
All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. Today we feature a rare treat, an … Continue reading Everything you wanted to know about COVID vaccines but never got a chance to ask
Month of CIRM: Making sure stem cell therapies don’t get lost in Translation
All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. Today we feature a blog written by … Continue reading Month of CIRM: Making sure stem cell therapies don’t get lost in Translation
How a CIRM scholar helped create a life-saving COVID vaccine
Dr. Derrick Rossi might be the most famous man whose name you don’t recognize. Dr. Rossi is the co-founder of Moderna. Yes, that Moderna. The COVID-19 vaccine Moderna. The vaccine that in clinical trials proved to be around 95 percent effective against the coronavirus. Dr. Rossi also has another claim to fame. He is a … Continue reading How a CIRM scholar helped create a life-saving COVID vaccine
Progress in the fight against Sickle Cell Disease
Marissa Cors, sickle cell disease patient advocate Last November Marissa Cors, a patient advocate in the fight against Sickle Cell Disease (SCD), told the Stem Cellar “A stem cell cure will end generations of guilt, suffering, pain and early death. It will give SCD families relief from the financial, emotional and spiritual burden of caring … Continue reading Progress in the fight against Sickle Cell Disease
The Stem Cellar 