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Marguerite Casillas gives patients a voice to make science stronger

Featured / 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 

Featured / 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

Featured / 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

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Summer of science for these CIRM students

Featured / California Institute for Regenerative Medicine (CIRM)

In a summer doubleheader, the California Institute for Regenerative Medicine (CIRM) hosted back-to-back conferences over two weekends for hundreds of aspiring California student scientists.  Together, these events highlight the pipeline of homegrown talent that will shape the next decade of stem cell and gene therapy breakthroughs in California. California voters invested in regenerative medicine not just to … Continue reading Summer of science for these CIRM students

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Three CIRM-funded approaches advance potential Parkinson’s treatments

Featured / California Institute for Regenerative Medicine (CIRM)

Holding a curved device that looked a bit like a mariner’s sextant, neurosurgeon Warren Boling explained how he’ll use it to precisely deliver a new gene therapy deep into the brains of Parkinson's patients participating in a pivotal clinical trial. A colony of iPSCs from a Parkinson's patient (left) and dopaminergic neurons made from these … Continue reading Three CIRM-funded approaches advance potential Parkinson’s treatments

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Experimental treatment brings relief for a dancer with chronic knee pain

Featured / California Institute for Regenerative Medicine (CIRM)

From the age of three, Tara Radcliffe Ghiglieri danced almost every day, leaping, pirouetting, tapping, and high-kicking her way into a Broadway career that included a multi-year stint as a Radio City Rockette. She trained hard, at times rehearsing eight hours a day, six days a week. "And performance days could be up to six … Continue reading Experimental treatment brings relief for a dancer with chronic knee pain

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Early trial shows promise for one‑shot cure for HIV 

Featured / California Institute for Regenerative Medicine (CIRM)

By Holly Alyssa MacCormick  A single infusion of a modified CAR-T cell therapy could pave the way for a potential one-shot cure for HIV, according to preliminary findings of a small, early-stage clinical trial. Steven G. Deeks, MD, the principal investigator of this California Institute for Regenerative Medicine (CIRM)-funded clinical trial, discussed the findings at a recent CIRM board meeting.  Steven Deeks, MD Photo: UCSF Roughly 40 million people worldwide and more … Continue reading Early trial shows promise for one‑shot cure for HIV 

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Genascence discusses gene therapy for knee osteoarthritis

Featured / California Institute for Regenerative Medicine (CIRM)

Knee joint pain rarely gets counted among major public‑health burdens. Yet it affects more than 3 million Californians and 34 million people across the U.S., with both massive economic and health consequences. Costly Impact In California alone, the cost of knee replacement surgeries exceeds $8 billion annually, according to Thomas Chalberg, PhD, founder and CEO … Continue reading Genascence discusses gene therapy for knee osteoarthritis

How California Built a Global Powerhouse in Stem Cell and Gene Therapy 

Featured / California Institute for Regenerative Medicine (CIRM)

A decision California voters made more than two decades ago helped build an unparalleled scientific ecosystem that's now paying dividends for the state, according to Johnathan Thomas, the president and CEO of the California Institute for Regenerative Medicine (CIRM). "(California) is pretty much now the largest funder of stem cell and gene therapy research in the world," … Continue reading How California Built a Global Powerhouse in Stem Cell and Gene Therapy 

Funding Available to Create Software to Integrate Regenerative Medicine Data

/ California Institute for Regenerative Medicine (CIRM)

Scientific breakthroughs are built on insights from good data, but sometimes those breakthroughs are hidden under an ocean of too much of it.   Integrating data Recently, the California Institute for Regenerative Medicine (CIRM) approved a new kind of investment. A new infrastructure award aims to create tools to make sense of the massive, complex biological datasets produced through CIRM-funded research. The program would fund the creation of new … Continue reading Funding Available to Create Software to Integrate Regenerative Medicine Data

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