New gene therapy aims to restore function in early-onset Parkinson’s

Image source: Shutterstock Nearly one million people in the U.S. are living with Parkinson’s disease, which causes progressive symptoms including tremors, stiffness, and gait and balance issues. Although there are medications to treat symptoms, the disease has no cure.  To date, the California Institute for Regenerative Medicine (CIRM) has invested more than $109 million in … Continue reading New gene therapy aims to restore function in early-onset Parkinson’s

How a childhood condition sparked a career in regenerative medicine

CIRM Scholar Dongwei Sun, PhD (left), with his mentor Huinan Liu, PhD, professor of bioengineering at the University of California, Riverside. As a child, Dongwei Sun underwent a medical treatment that left his lower limbs weak and occasionally numb. "This inspired my dedication to develop biomedical technologies that can prevent, treat, and potentially cure debilitating … Continue reading How a childhood condition sparked a career in regenerative medicine

Diving into a new era of treatment: A patient’s journey with an epilepsy stem cell therapy

Justin Graves diving by the Grand Cayman Islands In 2007, Justin Graves was working at a SCUBA shop in Louisville, Kentucky, when he had his first epileptic seizure. "SCUBA diving has just been something I've loved my whole life," he said. "My whole family loves it." Justin had to give up diving as well as … Continue reading Diving into a new era of treatment: A patient’s journey with an epilepsy stem cell therapy

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. This has been true since CIRM was founded in 2004, and federal funding for embryonic stem cell research was extremely hard to come by. Now, given current … Continue reading Filling gaps in federal research funding with a focus on neurological diseases 

20 Years of CIRM: From building the infrastructure to clinical trials 

Twenty years ago, Californians voted in favor of Proposition 71, which allocated $3 billion to support stem cell research and created the California Institute for Regenerative Medicine (CIRM). In 2020, Proposition 14 passed, providing an additional $5.5 billion. CIRM will reflect on the past two decades, highlighting early research that has evolved into potential therapies, … Continue reading 20 Years of CIRM: From building the infrastructure to clinical trials 

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

A CIRM Bridges student discovers a passion for engineering 

CIRM Bridges student Esme Ruiz-Garcia Esmerelda “Esme” Ruiz-Garcia grew up in California in a Mexican-American family, where pursuing a career in healthcare is considered a family calling, with most of her relatives in the nursing and medical fields. “I’ve always loved science, but I was not that interested in the medical field itself,” said Esme. … Continue reading A CIRM Bridges student discovers a passion for engineering 

CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders 

Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) announced in January $100 million in funding for a broad range of projects, including four to support clinical programs.  One of these was a $2.8 million award to Patricio Sepulveda, PhD, MBA, CEO of Amplo Biotechnology, Inc. That grant will support the development of a gene … Continue reading CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders 

Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment

A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasn’t right. But it wasn’t until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis – a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment

Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC

Histology of human cardiac muscle under microscope. Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) recently announced funding a series of projects spanning the earliest stages of research through to clinical trials. One of those projects was an $8 million award to Matthew Pollman, MD, MS, Senior Vice President of Clinical Development at Tenaya … Continue reading Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC