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
gene therapy
Genascence discusses gene therapy for knee osteoarthritis
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
CIRM funded clinical trial gives siblings a second chance at life
written by Holly Alyssa MacCormick Image Credit: The Langenhop Family Alicia Langenhop was seven months pregnant with her third child when she and her husband, Jon, learned that their two toddlers had a life-threatening immune disease called leukocyte adhesion deficiency-1 (LAD-1). One in a million LAD-1 is extremely rare—about one in a million births—but because … Continue reading CIRM funded clinical trial gives siblings a second chance at life
Elpida Therapeutics’ Study on Rare Nerve Disease CMT4J
CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth disease type 4J (CMT4J).
UC San Diego Gene Therapy Initiative Symposium fueling progress in the field
Enthusiasm ran high at the inaugural UC San Diego Gene Therapy Initiative Symposium at the Roth Auditorium at the Sanford Consortium. Researchers, clinicians, and patient advocates gathered to share new discoveries in gene therapy—a rapidly evolving field that is reshaping modern medicine. CIRM served as a platinum sponsor of the event. Era of progress The … Continue reading UC San Diego Gene Therapy Initiative Symposium fueling progress in the field
CIRM Supports Gene Therapy for Rare Heart Disorder
Image Source: American Heart Association Journals The California Institute for Regenerative Medicine (CIRM) awarded $4 million to support a gene therapy for rare life-threatening genetic disorder. A rare heart disorder Known as desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), the genetic disorder is a rare heart disorder. This preclinical award went to Deborah D. Ascheim, … Continue reading CIRM Supports Gene Therapy for Rare Heart Disorder
How a Genetic Therapy Helped Connor
Photo credit: n-Lorem Like many other 15 year-olds, Connor Dalby loves music and the beach, but unlike most of his peers his life has been anything but ordinary. As an infant he struggled with sleep and persistent stomach issues. As time went on, his condition worsened. By eight months old he was experiencing seizures, sometimes … Continue reading How a Genetic Therapy Helped Connor
Gene therapy trial tests cancer-killing virus in brain tumors
Dr. Noriyuki Kasahara of UCSF has received $11.8 million from CIRM to target newly diagnosed high‑grade gliomas. These aggressive brain and spinal cord cancers are extremely hard to treat. The grant supports research on DB107, formerly Toca 511, an innovative gene therapy that selectively targets tumor cells. The therapy delivers an enzyme that converts a … Continue reading Gene therapy trial tests cancer-killing virus in brain tumors
Equipping California nurses with skills to support cell and gene therapy patients
Regenerative medicine is a rapidly evolving field. As more therapies reach commercialization and clinical trial stages, there remains an immediate need to equip direct patient care nurses with skills to support patients on complex clinical trials as well as novel standard-of-care therapies. To address this need and continue efforts to build a regenerative medicine workforce … Continue reading Equipping California nurses with skills to support cell and gene therapy patients
Investing in a stem cell treatment for Hurler syndrome
The California Institute for Regenerative Medicine (CIRM) awarded $5,444,353 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This inherited condition is caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. … Continue reading Investing in a stem cell treatment for Hurler syndrome