THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a … Continue reading Creating a ‘bespoke’ approach to rare diseases
Rare Diseases
The bootcamp helping in the fight against rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Emil Kakkis at the Rare Entrepreneur Bootcamp Imagine you or someone you love is diagnosed with a rare disease and then told, “There is no cure, there are no treatments and because it’s so rare no one is even doing any research into developing a … Continue reading The bootcamp helping in the fight against rare diseases
Stem cell agency invests in therapy using killer cells to target colorectal, breast and ovarian cancers
While there have been some encouraging advances in treating cancer in recent decades, there are still many cancers that either resist treatment or recur after treatment. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved investing in a therapy targeting some of these hard-to-treat tumors. BioEclipse Therapeutics Inc. was awarded nearly … Continue reading Stem cell agency invests in therapy using killer cells to target colorectal, breast and ovarian cancers
Promoting stem cell therapies, racial justice, and fish breeding
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Jan Nolta, PhD, in her lab at UC Davis; Photo courtesy UC Davis Working at CIRM, you get to meet many remarkable people, and Jan Nolta, PhD, certainly falls into that category. Jan is the Director of the Stem Cell Program at UC Davis School of Medicine. She … Continue reading Promoting stem cell therapies, racial justice, and fish breeding
Rare Disease: An Uphill Battle for Diagnosis and Treatment
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST From left to right: Baby Dalia pre-diagnosis, Dalia on her way to the kindergarten, and Dalia today. When Dalia was 5 years old, she was finally diagnosed with MERRF syndrome- an extremely rare form of mitochondrial disease. By then, her parents had been searching for an … Continue reading Rare Disease: An Uphill Battle for Diagnosis and Treatment
Joining the movement to fight rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST It’s hard to think of something as being rare when it affects up to 30 million Americans and 300 million people worldwide. But the truth is there are more than 6,000 conditions – those affecting 200,000 people or fewer – that are considered rare. Today, … Continue reading Joining the movement to fight rare diseases
CIRM-supported therapy for blood cancers gets FDA fast track
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST People often complain about how long it can take to turn a scientific discovery into an approved therapy for patients. And they’re right. It can take years, decades even. But for Immune-Onc Therapeutics the path to FDA approval may just have been shortened. Back in April … Continue reading CIRM-supported therapy for blood cancers gets FDA fast track
Two Early-Stage Research Programs Targeting Cartilage Damage Get Funding from Stem Cell Agency
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Darryl D'Lima: Scripps Health Every year millions of Americans suffer damage to their cartilage, either in their knee or other joints, that can eventually lead to osteoarthritis, pain and immobility. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two projects targeting … Continue reading Two Early-Stage Research Programs Targeting Cartilage Damage Get Funding from Stem Cell Agency
UCLA gene therapy offers children with LAD-1 a new chance at living a normal life
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Photo courtesy of Tamara Hogue/UCLA Broad Stem Cell Research Center Leukocyte adhesion deficiency type 1 (LAD-1) is a rare pediatric disorder that causes the immune system to malfunction, resulting in recurrent, often severe, bacterial and fungal infections as well as delayed wound healing. This is because of a missing protein that would normally … Continue reading UCLA gene therapy offers children with LAD-1 a new chance at living a normal life
Looking back and looking forward: good news for two CIRM-supported studies
https://open.spotify.com/episode/4IimCtQ8STpFikwZRXqSAB Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about CIRM-funded research targeting blood … Continue reading Looking back and looking forward: good news for two CIRM-supported studies
The Stem Cellar 