Dr. Stephanie Cherqui Last week saw a flurry of really encouraging reports from projects that CIRM has supported. We blogged about two of them last Wednesday, but here’s another two programs showing promising results. UC San Diego researcher Dr. Stephanie Cherqui is running a CIRM-funded clinical trial for cystinosis. This is a condition where patients … Continue reading Two rare diseases, two pieces of good news
Gene editing
Living proof science can find a cure
Like many kids, let’s face it, many adults too, Ronav “Ronnie” Kashyap is getting a little bored stuck inside all day during the coronavirus pandemic. This video, shot by his dad Pawash, shows Ronnie trying to amuse himself by pretending to be hard at work. https://www.instagram.com/p/B_BSQaonFXb/ It’s a lovely moment. It’s also a moment that … Continue reading Living proof science can find a cure
CIRM-funded treatment for Cystinosis receives orphan drug designation
Dr. Stephanie Cherqui, UC San Diego Orphan drug designation is a special status given by the Food and Drug Administration (FDA) for potential treatments of rare diseases that affect fewer than 200,000 in the U.S. This type of status can significantly help advance treatments for rare diseases by providing financial incentives in the form of … Continue reading CIRM-funded treatment for Cystinosis receives orphan drug designation
‘A Tornado at the Front Door, a Tsunami at the Back Door’
CIRM funds a lot of research and all of it has life-saving potential. But every once in a while you come across a story about someone benefiting from CIRM-supported research that highlights why the work we do is so important. This story is about a brilliant researcher at UC San Diego developing a treatment for … Continue reading ‘A Tornado at the Front Door, a Tsunami at the Back Door’
Stem Cell/Gene Therapy combo heals patients battling rare disorder
Brenden Whittaker and his dog: Photo by Colin McGuire A few years ago, Brenden Whittaker was running out of time. Brenden was born with a rare condition called x-linked chronic granulomatous disease or XCGD. It meant he lacked a critical part of his immune system that protects against bacterial or fungal infections. Over 22 years … Continue reading Stem Cell/Gene Therapy combo heals patients battling rare disorder
Two studies identify a molecule that could be used to block Zika virus and kill cancer cells
Dr. Tariq Rana (left) and Dr. Jeremy Rich (right) both lead independent teams at UC San Diego that identified a molecule, αvβ5 integrin, as the Zika virus' key to getting into brain stem cells Zika virus is caused by a virus transmitted by Aedes mosquitoes. People usually develop mild symptoms that include fever, rash, and … Continue reading Two studies identify a molecule that could be used to block Zika virus and kill cancer cells
CIRM supported study of gene silencer blocks ALS degeneration, saves motor function
Dr. Martin Marsala, UC San Diego Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a neurodegenerative disease that destroys the nerve cells in the brain and spinal cord. As a result of ALS, the motor neurons that enable bodily movement and muscle control are harmed, which can make it difficult to move, … Continue reading CIRM supported study of gene silencer blocks ALS degeneration, saves motor function
The Top CIRM Blogs of 2019
This year the most widely read blog was actually one we wrote back in 2018. It’s the transcript of a Facebook Live: “Ask the Stem Cell Team” event about strokes and stroke recovery. Because stroke is the third leading cause of death and disability in the US it’s probably no surprise this blog has lasting … Continue reading The Top CIRM Blogs of 2019
Facebook Live: Ask the Stem Cell Team
On December 12th we hosted our latest 'Facebook Live: Ask the Stem Cell Team' event. This time around we really did mean team. We had a host of our Science Officers answering questions from friends and supporters of CIRM. We got a lot of questions and didn't have enough time to address them all. So … Continue reading Facebook Live: Ask the Stem Cell Team
CIRM’s Alpha Stem Cell Clinics Given High Profile Role in Clinical Trials Network
Sue and Bill Gross Hall Photo by Hoang Xuan Pham/ UC Irvine There are a growing number of predatory clinics in California and around the US, offering unproven stem cell therapies. For patients seeking a legitimate therapy it can often be hard finding a reliable clinic, one offering treatments based on the rigorous science required … Continue reading CIRM’s Alpha Stem Cell Clinics Given High Profile Role in Clinical Trials Network
The Stem Cellar 