Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) announced in January $100 million in funding for a broad range of projects, including four to support clinical programs. One of these was a $2.8 million award to Patricio Sepulveda, PhD, MBA, CEO of Amplo Biotechnology, Inc. That grant will support the development of a gene … Continue reading CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders
Muscular Dystrophy
A personal reason to develop a better gene therapy
THIS BLOG IS ALSO AVAILABLE AS AN AUDIOCAST ON SPOTIFY Credit : Allison Dougherty, Broad Communications For Sharif Tabebordbar, finding a gene therapy for genetic muscle wasting diseases was personal. When he was a teenager, his father was diagnosed with a rare genetic muscle disease that eventually left him unable to walk. In an interview … Continue reading A personal reason to develop a better gene therapy
Repairing damaged muscles
Close-up of the arm of a 70-year-old male patient with a torn biceps muscle as a result of a bowling injury; Photo courtesy Science Photo Library In the time of coronavirus an awful lot of people are not just working from home they’re also working out at home. That’s a good thing; exercise is a … Continue reading Repairing damaged muscles
Perseverance: from theory to therapy. Our story over the last year – and a half
Some of the stars of our Annual Report It’s been a long time coming. Eighteen months to be precise. Which is a peculiarly long time for an Annual Report. The world is certainly a very different place today than when we started, and yet our core mission hasn’t changed at all, except to spring into … Continue reading Perseverance: from theory to therapy. Our story over the last year – and a half
Deep dive into muscle repair yields new strategies to combat Duchenne muscular dystrophy
Researchers at the Sanford Burnham Prebys Medical Discovery Institute (SBP) reported new findings this week that may lead to novel therapeutic strategies for people suffering from Duchenne muscular dystrophy (DMD). DMD, a muscle-wasting disease that affects 1 in 7250 males aged 5 to 24 years in the United States, is caused by a genetic mutation leading … Continue reading Deep dive into muscle repair yields new strategies to combat Duchenne muscular dystrophy
A scalable, clinic-friendly recipe for converting skin cells to muscle cells
Way back in 1987, about two decades before Shinya Yamanaka would go on to identify four proteins that can reprogram skin cells into induced pluripotent stem cells (iPSCs), Harold Weintraub’s lab identified the first “master control” protein, MyoD, which can directly convert a skin cell into a muscle cell. Though MyoD opened up new approaches … Continue reading A scalable, clinic-friendly recipe for converting skin cells to muscle cells
Stem Cell Roundup: Improving muscle function in muscular dystrophy; Building a better brain; Boosting efficiency in making iPSC’s
Here are the stem cell stories that caught our eye this week. Photos of the week TGIF! We’re so excited that the weekend is here that we are sharing not one but TWO amazing stem cell photos of the week. Photo #1 is borrowed from a blog we wrote earlier this week about a new … Continue reading Stem Cell Roundup: Improving muscle function in muscular dystrophy; Building a better brain; Boosting efficiency in making iPSC’s
Stem Cell Stories that Caught Our Eye: GPS for Skin & Different Therapies for Aging vs. Injured Muscles?
Skin stem cells specialize into new skin by sensing neighborhood crowding When embarking on a road trip, the GPS technology inside our smartphones helps us know where we are and how to get where we’re going. The stem cells buried in the deepest layers of our skin don’t have a GPS and yet, they do … Continue reading Stem Cell Stories that Caught Our Eye: GPS for Skin & Different Therapies for Aging vs. Injured Muscles?
Stem Cell Stories That Caught our Eye: Stem Cell Therapies for Stroke and Duchenne Muscular Dystrophy Patients
With the Thanksgiving holiday behind us, we’re back to the grind at CIRM. Here are two exciting CIRM-funded stem cell stories that happened while you were away. Stanford Scientists Are Treating Stroke Patients with Stem Cells Smithsonian Magazine featured the work of a CIRM-funded scientist in their December Magazine issue. The article, “A Neurosurgeon’s Remarkable … Continue reading Stem Cell Stories That Caught our Eye: Stem Cell Therapies for Stroke and Duchenne Muscular Dystrophy Patients
Raising awareness about Rare Disease Day
One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases. Since then we have funded 13 new clinical trials including four targeting rare diseases (retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s … Continue reading Raising awareness about Rare Disease Day
The Stem Cellar 