Dr. Paul Szabolcs: Photo courtesy of UPMC A simple blood stem cell transplant is showing tremendous promise in treating a wide range of metabolic, blood and immune disorders such as thalassemia and some leukodystrophies. These are considered rare diseases – meaning there are fewer than 200,000 people with them in the US – so there … Continue reading Cord blood transplants help children fighting deadly diseases
Rare Diseases
A Tribute to Huntington’s Disease Warriors in the Age of COVID-19
Frances Saldana is one of the most remarkable women I know. She has lost all three of her children to Huntington's disease (HD) - a nasty, fatal disease that steadily destroys the nerve cells in the brain - but still retains a fighting spirit and a commitment to finding a cure for HD. She is … Continue reading A Tribute to Huntington’s Disease Warriors in the Age of COVID-19
A clear vision for the future
Dr. Henry Klassen and Dr. Jing Yang, founders of jCyte When you have worked with a group of people over many years the relationship becomes more than just a business venture, it becomes personal. That's certainly the case with jCyte, a company founded by Drs. Henry Klassen and Jing Yang, aimed at finding a cure … Continue reading A clear vision for the future
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
Stem cell transplant in utero offers potential treatment for congenital diseases
Dr. Tippi Mackenzie, UCSFImage Credit: UCSF Each year, around 24,000 women in the US lose a pregnancy. One reason for this unfortunate occurrence are metabolic disorders, one of which is known as Sly syndrome and is caused by a single genetic mutation. In Sly syndrome, the body's cells lack an enzyme necessary for proper cell … Continue reading Stem cell transplant in utero offers potential treatment for congenital diseases
CIRM Board Meeting Highlights Important Updates to Clinical Trials
Dr. Maria T. Millan, President and CEO of CIRM, presenting the President's Report This past Thursday the governing Board of the California Institute for Regenerative Medicine (CIRM) were presented with an update on CIRM’s clinical portfolio, which to date includes 60 clinical trials in various areas including kidney failure, cancer, and other rare diseases. The … Continue reading CIRM Board Meeting Highlights Important Updates to Clinical Trials
‘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
It’s official: the state of regenerative medicine is strong
A panel discussion to a packed house at the annual ARM State of the Industry briefing Calling it a “year of dramatic impact for patients” Janet Lambert, the CEO of the Alliance for Regenerative Medicine (ARM), gave the annual “State of the Industry” briefing today in San Francisco. The briefing is a traditional kick-off event … Continue reading It’s official: the state of regenerative medicine is strong
The Stem Cellar 