Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle…
We propose a cellular trial operation at UCSF that will focus on childhood hereditary hematological and immunologic disorders and in anti-inflammatory lung cellular therapies. This alpha clinic operation at UCSF…
We propose a cellular trial operation at UCSF that will focus on childhood hereditary hematological and immunologic disorders and in anti-inflammatory lung cellular therapies. This alpha clinic operation at UCSF…
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 Patient specific stem cells (iPSCs) to model ALS and identify a broadly acting therapeutic intervention Impact Heterogenous patient population, dire need for broadly acting therapeutic interventions Major Proposed…
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…
Research Objective Use of a viral vector to transport a normal gene to children with severe motor and respiratory disabilities caused by an inborn defect of the choline acetyltransferase (ChAT)…
Research Objective The objective of this research proposal is to develop a lead AAV candidate for an optogenetic vision restoration therapy for patients suffering from blindness due to loss of…
Research Objective Genetically modified cells that help control immune reactions Impact Improved treatment for patients undergoing transplantation Major Proposed Activities To identify the optimal source of regulatory T cells for…
Get the latest CIRM news, information on upcoming meetings, and funding opportunities delivered to your inbox.
