Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease that prevents patients from combating infections. This leads to recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations, and can be fatal. It is caused by a mutation in a specific gene that causes low levels of a protein called CD18. The … Continue reading Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease
Blood stem cells
CIRM funding helps improve immune cell therapy to combat HIV
Image description: T cell infected with HIV. Image Credit: National Institute of Allergy and Infectious Diseases (NIAID) In June of last year we wrote about how Dr. Scott Kitchen and his team at UCLA are engineering blood forming stem cells in order to fight HIV, a potentially deadly virus that attacks the immune system and … Continue reading CIRM funding helps improve immune cell therapy to combat HIV
Three UC’s Join Forces to Launch CRISPR Clinical Trial Targeting Sickle Cell Disease
Sickle shaped red blood cells The University of California, San Francisco (UCSF), in collaboration with UC Berkeley (UCB) and UC Los Angeles (UCLA), have been given permission by the US Food and Drug Administration (FDA) to launch a first-in-human clinical trial using CRISPR technology as a gene-editing technique to cure Sickle Cell Disease. This research … Continue reading Three UC’s Join Forces to Launch CRISPR Clinical Trial Targeting Sickle Cell Disease
Prime Time for Rocket
Rocket Pharmaceuticals, a company that specializes in developing genetic therapies for rare childhood disorders, just got a big boost from the European Medicines Agency (EMA). They were given a Priority Medicines (PRIME) designation for their therapy for Leukocyte Adhesion Deficiency-1 (LAD-1). CIRM is funding ($6.56 million) Rocket’s clinical trial for LAD-I, an immune disorder that … Continue reading Prime Time for Rocket
Going the extra mile to save a patient’s life
You can tell an awful lot about a company by the people it hires and the ability it gives them to do their job in an ethical, principled way. By that measure Rocket Pharma is a pretty darn cool company. Rocket Pharma is running a CIRM-funded clinical trial for Leukocyte Adhesion Deficiency-I (LAD-I), a rare … Continue reading Going the extra mile to save a patient’s life
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
A word from our Chair, several in fact
In 2005, the New Oxford American Dictionary named “podcast" its word of the year. At the time a podcast was something many had heard of but not that many actually tuned in to. My how times have changed. Now there are some two million podcasts to chose from, at least according to the New York … Continue reading A word from our Chair, several in fact
Biotechnology companies join forces in developing treatment for X-SCID
Jasper Therapeutics, Inc., a biotechnology company focused on blood stem cell therapies, and Graphite Bio, Inc., a biotechnology company focused on gene editing therapies to treat or cure serious diseases, announced a research and clinical collaboration for a treatment for X-SCID. X-SCID, which stands for X-linked severe combined immunodeficiency, is a genetic disorder that interferes … Continue reading Biotechnology companies join forces in developing treatment for X-SCID
Surviving with Joy
Dr. Tippi MacKenzie (left) of UCSF Benioff Children’s Hospital San Francisco, visits with newborn Elianna and parents Nichelle Obar and Chris Constantino. Photo by Noah Berger Alpha thalassemia major is, by any stretch of the imagination, a dreadful, heart breaker of a disease. It's caused by four missing or mutated genes and it almost always … Continue reading Surviving with Joy
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 