Ayaan Isaacs was born in South Africa on March 4th, 2016 as a seemingly healthy baby. But only a few days in to life, he contracted a life-threatening liver infection. He thankfully survived, only to have the doctors discover a few weeks later that he had something much more troubling – a rare disease that … Continue reading The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Immune Disease
Friday Roundup: A better kind of blood stem cell transplant; Encouraging news from spinal cord injury trial; Finding an “elusive” cell that could help diabetics
Cool Instagram image of the week: Chemo- and radiation-free blood stem cell transplant showing promise Bubble baby disease, also known as severe combined immunodeficiency (SCID), is an inherited disorder that leaves newborns without an effective immune system. Currently, the only approved treatment for SCID is a blood stem cell transplant, in which the patient’s defective … Continue reading Friday Roundup: A better kind of blood stem cell transplant; Encouraging news from spinal cord injury trial; Finding an “elusive” cell that could help diabetics
Using the AIDS virus to help children battling a deadly immune disorder
More than 35 million people around the world have been killed by HIV, the virus that causes AIDS. So, it’s hard to think that the same approach the virus uses to infect cells could also be used to help children battling a deadly immune system disorder. But that’s precisely what researchers at UC San Francisco … Continue reading Using the AIDS virus to help children battling a deadly immune disorder
Progress to a Cure for Bubble Baby Disease
Welcome back to our “Throwback Thursday” series on the Stem Cellar. Over the years, we’ve accumulated an arsenal of exciting stem cell stories about advances towards stem cell-based cures for serious diseases. Today we’re featuring stories about the progress of CIRM-funded clinical trials for the treatment of a devastating, usually fatal, primary immune disease that … Continue reading Progress to a Cure for Bubble Baby Disease
Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]
During this second week of the Month of CIRM, we’ve been focusing on the people who are critical to accomplishing our mission to accelerate stem cell treatments to patients with unmet medical needs. These folks include researchers, like Clive Svendsen and his team at Cedars-Sinai Medical Center who are working tirelessly to develop a stem … Continue reading Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]
CIRM-Funded Clinical Trials Targeting Blood and Immune Disorders
This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs. This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Today we are featuring trials in our blood and … Continue reading CIRM-Funded Clinical Trials Targeting Blood and Immune Disorders
Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
There were lots of CIRM mentions in the news this week. Here are two brief recaps written by Karen Ring to get you up to speed. A third story by Todd Dubnicoff summarizes an promising finding related to heart disease by researchers in Singapore. CIRM-funded “bubble baby” disease therapy gets special designation by UK. … Continue reading Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
Stem cell stories that caught our eye: skin grafts fight diabetes, reprogramming the immune system, and Asterias expands spinal cord injury trial sites
Here are the stem cell stories that caught our eye this week. Skin grafts fight diabetes and obesity. An interesting new gene therapy strategy for fighting type 1 diabetes and obesity surfaced this week. Scientists from the University of Chicago made genetically engineered skin grafts that secrete a peptide hormone called glucagon-liked peptide-1 (GLP-1). This … Continue reading Stem cell stories that caught our eye: skin grafts fight diabetes, reprogramming the immune system, and Asterias expands spinal cord injury trial sites
CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
If you have read our 2016 Annual Report (and if you haven’t you should, it’s brilliant) or just seen the cover you’ll know that it features very prominently a young girl named Evie Padilla Vaccaro. Evie was born with Severe Combined Immunodeficiency or SCID – also known as “bubble baby disease”; we’ve written about it … Continue reading CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
Stem cell stories that caught our eye: new baldness treatments?, novel lung stem cells, and giraffe stem cells
Novel immune system/stem cell interaction may lead to better treatments for baldness. When one thinks of the immune system it’s usually in terms of the body’s ability to fight off a bad cold or flu virus. But a team of UCSF researchers this week report in Cell that a particular cell of the immune system is … Continue reading Stem cell stories that caught our eye: new baldness treatments?, novel lung stem cells, and giraffe stem cells