Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…
Research Objective With a single administration to an individual, lentiviral vectors that selectively transduce B cells in vivo and express highly potent anti-HIV-1 proteins to suppress HIV-1 replication throughout life…
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
Research Objective This personalized chimeric antigen receptor (CAR) T cell therapy will attack a wider range of cancer cells, and block cancer's defense mechanisms to empower the immune system to…
Research Objective Genome-edited autologous regulatory T cells for promoting immune tolerance in combination with T-cell-targeting therapies Impact We focus on type 1 diabetes in this proof-of-concept study, but the therapeutic…
Research Objective Allogeneic Human induced pluripotent stem cells-derived inhibitory interneuron therapy product encapsulated in a hyaluronan/VEGF nanoparticle hydrogel for the treatment of stroke Impact Previous stem cell-based technologies had poor…
Research Objective Allogeneic human induced pluripotent stem cell-derived glial enriched progenitor cell therapy to treat mild traumatic brain injury Impact Prior to this study, glial cell-based therapies have never been…
Research Objective Our goal is to use stem cell models to identify a small molecule drug candidate for GI motility disorders. Impact The candidate will be used for severe gastrointestinal…
Research Objective The research proposed here will support the development of NGL-101, a cell therapy for treating adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). Impact Successful development of…
Research Objective We aim to discover an adeno-associated viral (AAV) CRISPRi gene therapy for amyotrophic lateral sclerosis (ALS) patients with hexanucleotide repeat expansions in the C9orf72 gene. Impact This therapy…
Get the latest CIRM news, information on upcoming meetings, and funding opportunities delivered to your inbox.
