Therapeutic/Technology: Gene Therapy (All)


Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis

Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS disease phenotypes Impact Our goal is to determine if ataxin-3 genetic dysregulation is a target for the development of therapies to treat ALS (Lou Gehrig's […]

Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies

Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]

Development of universal off-the-shelf iPSC derived dendritic cells for use as patient specific anti-tumor vaccine

Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the genetics of the patient and their cancer. Impact Success would provide proof-of-principle of a novel cell based therapy for cancers broadly. It is expected to […]

Battling COVID-19 Using Off-The-Shelf HSC-Engineered iNKT Cells

Research Objective allogeneic HSC-engineered iNKT (HSC-iNKT) cells Impact treatment for COVID-19 Major Proposed Activities Milestone 1. Production of AlloHSC-iNKT and UHSC-iNKT cells Milestone 2. Characterization of the AlloHSC-iNKT and UHSC-iNKT cells Milestone 3. Delivery of the new therapeutic candidate The novel SARS-CoV-2 is the cause of the coronavirus disease 19 (COVID-19) pandemic, which is responsible […]

Chimeric Antigen Receptor Targeting Spike Glycoprotein of SARS-cov2

Research Objective We expect to generate iPSC derived NK cells expressing a CAR against SARS-cov2 that could be used as an off-the-shelf therapy for COVID-19 Impact The proposed studies will provide a novel therapeutic approach to boost the cellular immunity against SARS-cov2, especially for high risk populations. Major Proposed Activities Construction of SARS-cov2 CAR constructs […]

Persistent Off-the-Shelf meACE2-CAR-IL-15 NK Cells Derived from CD34(+) Cord Blood Stem Cells to Prevent and Treat COVID-19

Research Objective To develop and characterize meACE-2-CAR-IL15 NK cells expressing a mutated ACE2 and IL-15, allowing specific killing of SARS-CoV-2-infected cells and long in vivo persistence of the engineered cells. Impact To provide a timely, novel, and effective cell therapy for COVID-19, which has no FDA-approved vaccines and only remdesivir has received an emergency-use approval. […]

Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

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 cell disease (adults and adolescents) with repeated, severe painful and lung events are eligible for the clinical trial Therapeutic Mechanism Blood stem cells are harvested […]

Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease

Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle globin and induction of anti-sickling fetal hemoglobin Unmet Medical Need Sickle cell is a severe disease with protean manifestations. The only curative therapy is an […]

Evaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8

University of California, San Francisco (UCSF) CIRM Alpha Stem Cell Clinic

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 will expand participation by California citizens in these novel clinical themes. The objective of this proposal from the Univ. of Calif., San Francisco is to […]

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