Recently, the California Institute for Regenerative Medicine (CIRM) announced a new approach for how its funding can advance treatments for people living with rare diseases. Individually, few people have any single rare disease, but in aggregate, about 30 million people in the U.S. have one of the roughly 10,000 rare diseases, and 95% have no … Continue reading New program to help patients with rare disease
Research
Early-stage research could benefit those with genetic diseases
Written by Holly Alyssa MacCormick Image Credit: Audrey Davidow－Calvin outside Wanting the best for your child is a universal experience, and Audrey Davidow wanted her baby to have a bright future full of possibility. But in March of 2012, her 13-month-old son Calvin was diagnosed with a rare disease called Pitt Hopkins Syndrome, and she … Continue reading Early-stage research could benefit those with genetic diseases
Fueling the Brain: Exploring the connection between metabolism and neurodevelopment
Top left to right: Aparna Bhaduri, PhD; Heather Christofk, PhD; Harley Kornblum, PhD; William Zeiger, MD, PhDBottom Left to right: Elizabeth Crouch, PhD; Mohammed Mostajo-Radji, PhD; Carrie Beardon, PhD; Michael Zito, MD As part of our goal of accelerating discoveries and therapies for central nervous system disorders, in 2023, the California Institute for Regenerative Medicine … Continue reading Fueling the Brain: Exploring the connection between metabolism and neurodevelopment
Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD
Image Credit: The Adler Lab at the University of California, San Diego In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD
Gene therapies for Friedreich’s Ataxia: A conversation with Liz Soragni, PhD
Liz Soragni, PhD, Director of Research at the Friedreich’s Ataxia Research Alliance In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene therapies for Friedreich’s Ataxia: A conversation with Liz Soragni, PhD
20 years of CIRM: After spurring new research, CIRM’s iPSC repository closes
In celebration of CIRM’s 20th anniversary, we have been reflecting on some of the early projects we supported and their outcomes. This blog is a little different, because it also marks the end of a project. Stem cell lines In 2013 CIRM announced a $32 million project to create a repository of stem cell lines … Continue reading 20 years of CIRM: After spurring new research, CIRM’s iPSC repository closes
Filling gaps in federal research funding with a focus on neurological diseases
Over the past 20 years, the California Institute for Regenerative Medicine (CIRM) has supercharged research in California in ways that have often filled gaps in federal research funding and most recently by focusing on gaps in neurological disease. A few different factors are limiting biomedical research funding across the United States. In addition to recent … Continue reading Filling gaps in federal research funding with a focus on neurological diseases
How Patient-Derived Cells Are Changing ALS Research
Research Investigator Julia Kaye, PhD, stands for a portrait in a lab at Gladstone Institutes. Photo courtesy of Gladstone Institutes. Before there can be any therapies for disease, scientists need to understand what goes wrong in the first place. Are cells lost that could be replaced? Is there too much or too little of a … Continue reading How Patient-Derived Cells Are Changing ALS Research
The Link Between Air Pollution and Autism
The California Institute for Regenerative Medicine (CIRM) is supporting innovative research into neuro diseases, including a project examining how air pollution may contribute to autism. Through the Research using Multidisciplinary, Innovative approaches in Neuro Diseases (ReMIND) program, Stuart Lipton, MD, PhD, and co-investigators Tomo Nakamura, PhD, John Yates, PhD, Marisa Roberto, PhD, and Nik Schork, … Continue reading The Link Between Air Pollution and Autism
Using Genetic Sequencing and AI to Identify and Treat Schizophrenia
Top left to right: Tomasz Nowakowski, PhD, and Nevan Krogan, PhD Bottom left to right: Aparna Bhaduri, PhD; Matt State, PhD; Alice Ting, PhD; Trey Ideker, PhD; Willow Coyote-Maestas, PhD A UC San Francisco research team funded by the California Institute for Regenerative Medicine (CIRM) is using genetic sequencing and AI to identify and treat … Continue reading Using Genetic Sequencing and AI to Identify and Treat Schizophrenia