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 Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
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 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 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 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…
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