New gene therapy for 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.  CIRM's support for potential Parkinson's treatments To date, the California Institute for Regenerative Medicine (CIRM) has … Continue reading New gene therapy for 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 received a medical treatment that left his lower limbs weak and sometimes numb. “This experience inspired my dedication to develop biomedical technologies to prevent, treat, and potentially cure debilitating … Continue reading How a childhood condition sparked a career in regenerative medicine

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." Medication not always effective Justin had to give up … Continue reading 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 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 

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

Californians voted to support stem cell research 20 years ago. Now we’re building on what we’ve created through the California Institute for Regenerative Medicine (CIRM). Their initial $3 billion investment through Proposition 71 paved the way for voters to approve Proposition 14 in 2020. That added another $5.5 billion to advance the science. CIRM will … 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

CIRM Bridges Program: Cultivating Future Scientists

Esmerelda “Esme” Ruiz-Garcia grew up in California in a Mexican-American family, where pursuing a career in healthcare is considered a family calling, but through CIRM's Bridges program she's now pursuing her Ph.D. focused on bioengineering and stem cell research.

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. A rare heart condition One of those projects was an $8 million award to Matthew Pollman, MD, MS, Senior Vice President of … Continue reading Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC