How mRNA and CRISPR-Cas9 could treat muscle atrophy

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Researchers use mRNA to introduce the gene editor CRISPR-Cas9 into human muscle stem cells. These cells fused into multinucleated myotubes following mRNA-mediated CRISPR-Cas9 gene editing. A myosin heavy chain is seen in green and the nuclei in blue. Photo: Spuler Lab A team of researchers from … Continue reading How mRNA and CRISPR-Cas9 could treat muscle atrophy

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

Board Funds Fifteen Bridges to Stem Cell Research and Therapy Programs Across California and New Sickle Cell Disease Trial

Yesterday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $8.39 million to the University of California, San Francisco (UCSF) to fund a clinical trial for sickle cell disease (SCD).  An additional $51.08 million was awarded to fifteen community colleges and universities across California to fund undergraduate and master’s level programs that … Continue reading Board Funds Fifteen Bridges to Stem Cell Research and Therapy Programs Across California and New Sickle Cell Disease Trial

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

CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial

Dr. Matthew Porteus The US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) permission enabling Graphite Bio to test the investigational, potentially revolutionary gene editing therapy GPH101 developed under the supervision of Matthew Porteus, MD, PhD, in a clinical trial for people with sickle cell disease (SCD). The California Institute for Regenerative … Continue reading CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial

CIRM progression award to support research towards immunodeficiency

Dr. Caroline Kuo, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA In 2017, CIRM funded a discovery or early stage research project for Dr. Caroline Kuo at UCLA for a hereditary immune disorder known as X-Linked Hyper IgM Syndrome. The work has gone so well … Continue reading CIRM progression award to support research towards immunodeficiency

Stanford scientist uses CRISPR-Cas9 and stem cells to develop potential “bubble baby” therapy

Dr. Matthew Porteus, professor of pediatrics at Stanford University. Photo courtesy of Stanford Medicine. Our immune system is an important and essential part of everyday life. It is crucial for fighting off colds and, with the help of vaccinations, gives us immunity to potentially lethal diseases. Unfortunately, for some infants, this innate bodily defense mechanism … Continue reading Stanford scientist uses CRISPR-Cas9 and stem cells to develop potential “bubble baby” therapy

CRISPR-Cas9 101: an overview and the role it plays in developing therapies

Illustration courtesy of TED website There has been a lot of conversation surrounding CRISPR-Cas9 in these recent months as well as many sensational news stories. Some of these stories highlight the promise this technology holds, while others emphasize a word of caution. But what exactly does this technology do and how does it work? Here … Continue reading CRISPR-Cas9 101: an overview and the role it plays in developing therapies

Midwest universities are making important tools to advance stem cell research

Two Midwest universities are making headlines for their contributions to stem cell research. Both are developing important tools to advance this field of study, but in two unique ways. Scientists at the University of Michigan (UM), have compiled an impressive repository of disease-specific stem cell lines. Cell lines are crucial tools for scientists to study … Continue reading Midwest universities are making important tools to advance stem cell research

For the first time, scientists entirely reprogram human skin cells to iPSCs using CRISPR

Back in 2012, Shinya Yamanaka was awarded the Nobel Prize in Physiology or Medicine for his group’s identification of “Yamanaka Factors,” a group of genes that are capable of turning ordinary skin cells into induced pluripotentent stem cells (iPSCs) which have the ability to become any type of cell within the body. Discovery of iPSCs … Continue reading For the first time, scientists entirely reprogram human skin cells to iPSCs using CRISPR