The California Institute for Regenerative Medicine (CIRM) awarded $5,444,353 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This is an inherited condition caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest … Continue reading Investing in a stem cell treatment for Hurler syndrome
Gene editing
Funding a Clinical Trial for a Functional Cure for HIV
The use of antiretroviral drugs has turned HIV/AIDS from a fatal disease to one that can, in many cases in the US, be controlled. But these drugs are not a cure. That's why the governing Board of the California Institute for Regenerative Medicine (CIRM) voted to approve investing $6.85 million in a therapy that aims … Continue reading Funding a Clinical Trial for a Functional Cure for HIV
Tiny tools for the smallest of tasks, editing genes
YOU CAN LISTEN TO THIS BLOG AS AN AUDIOCAST ON SPOTIFY Developing new tools to edit genes Having the right tools to do a job is important. Try using a large screwdriver to tighten the screws on your glasses and you quickly appreciate that it’s not just the type of tool that’s important, it’s also … Continue reading Tiny tools for the smallest of tasks, editing genes
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
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
Researcher claims to have made first gene-edited baby. But did it really happen?
Remember the Raelians? Probably not. But way back in 2002 the group, some described them as a cult, claimed it had created the world’s first cloned baby. The news made headlines all around the world raising fears we were stepping into uncharted scientific territory. Several weeks later the scientist brought in by the Raelians to … Continue reading Researcher claims to have made first gene-edited baby. But did it really happen?
Gene-editing Technique in Mice Shows Promise for Genetic Disorder in Utero
For first time ever, scientists used a gene editing technique to successfully cure a genetic condition in a mouse in utero. Their findings present a promising new avenue for research into treating genetic conditions in utero.
Coming up with a stem cell FIX for a life-threatening blood disorder
A promising new treatment option for hemophiliacs is in the works at the Salk Institute for Biological Sciences. Patients with Hemophilia B experience uncontrolled, and sometimes life threatening, bleeding due to loss or improper function of Factor IX (FIX), a protein involved in blood clotting. There is no cure for the disease and patients rely … Continue reading Coming up with a stem cell FIX for a life-threatening blood disorder
Stem Cell Roundup: New understanding of Huntington’s; how stem cells can double your DNA; and using “the Gary Oldman of cell types” to reverse aging
This week's roundup highlights how we are constantly finding out new and exciting ways that stem cells could help change the way we treat disease. Our Cool Stem Cell Image of the Week comes from our first story, about unlocking some of the secrets of Huntington's disease. It comes from the Laboratory of Stem Cell … Continue reading Stem Cell Roundup: New understanding of Huntington’s; how stem cells can double your DNA; and using “the Gary Oldman of cell types” to reverse aging
How a tiny patch of skin helped researchers save the life of a young boy battling a deadly disease
By any standards epidermolysis bullosa (EB) is a nasty disease. It’s a genetic condition that causes the skin to blister, break and tear off. At best, it’s painful and disfiguring. At worst, it can be fatal. Now researchers in Italy have come up with an approach that could offer hope for people battling the … Continue reading How a tiny patch of skin helped researchers save the life of a young boy battling a deadly disease
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