Research & Science

CIRM funds science research from the discovery stage through all phases of clinical trials.  We highlight new findings in gene and cell therapies, as well as technology and related science news.

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Marguerite Casillas gives patients a voice to make science stronger

/ California Institute for Regenerative Medicine (CIRM)

For about half an hour, Marguerite Casillas broke the spell cast by speakers discussing transcription factors, recombinant DNA, and other scientific breakthroughs.   She reminded an audience of undergraduate, graduate, and postdoctoral scientists at a California Institute for Regenerative Medicine (CIRM) trainee networking conference what their work is ultimately about.   "Behind every sample, every data point is a … Continue reading Marguerite Casillas gives patients a voice to make science stronger

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CAR T-cell therapy and how it’s used at CIRM 

/ California Institute for Regenerative Medicine (CIRM)

By Holly Alyssa MacCormick  The human body is remarkably adept at defending itself from harmful cells and foreign substances, but sometimes it needs a little help. One way that scientists boost the immune system is through a form of genetic engineering known as chimeric antigen receptor, or CAR T-cell therapy. This therapy is currently used to treat certain cancers, but it may soon be used to treat other diseases as well. The biology behind CAR T-cell therapy  T-cells are a special type of immune cell that works as a sentinel in the … Continue reading CAR T-cell therapy and how it’s used at CIRM 

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How patient advocates help advance profound autism research

/ California Institute for Regenerative Medicine (CIRM)

By Holly Alyssa MacCormick Some of the biggest advances in autism research have come from a unique and dedicated group—the families and patient advocates who live with autism every day. Matthew State, MD, PhD, chief scientific advisor for the Aligning Research to Impact Autism (ARIA) initiative and chair of psychiatry and behavioral sciences at the … Continue reading How patient advocates help advance profound autism research

Progress on Sickle Cell Disease Treatments 

/ California Institute for Regenerative Medicine (CIRM)

Only three years ago, the FDA approved the first gene therapies for sickle cell disease (Lyfgenia and Casvegy), historic breakthroughs decades in the making. Yet on World Sickle Cell Awareness Day this year, the reality is that high cost, limited availability, and complex logistics keep these treatments out of reach for most people who need them.   Yet these breakthroughs offer tremendous hope. For many years, the … Continue reading Progress on Sickle Cell Disease Treatments 

New program to help patients with rare disease

/ Amy Adams / 2 Comments

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

Early-stage research could benefit those with genetic diseases

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

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

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

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

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

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

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

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 

/ California Institute for Regenerative Medicine (CIRM) / 2 Comments

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