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99,167 grants found
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Modulation of Immune responses to gene therapy by Creation of AAV-specific Chimeric antigen receptor regulatory T cells (CAR-Tregs)
Project 4 - Abstract In Project 4, we will use our novel adeno-associated virus (AAV) directed chimeric antigen receptor (CAR) T-cells and T regulatory cells (Tregs) to modulate an...
Models and Gene Therapies for AAT Deficiency
Administrative Core Project Summary The administrative core will provide a structure for management of the financial aspects of this complex multi-component program and create v...
Vector Immunology Core
CORE A - SUMMARY/ABSTRACT: The Vector Immunology Core will directly support all four projects of this program project grant by providing reliable, consistent and standardized im...
Human Genetic Disease Modeling and Physiology Core
Core B - Project Summary To have successful therapies for Alpha-1 antitrypsin deficiency it is necessary to have animal models that recapitulate the phenotypes see in human patie...
Optimized Gene Replacement for AAT deficiency and Modeling of Clinical Outcomes in small and large animal models
Project Summary (Project 1) Alpha-1 antitrypsin deficiency (AATD) is a common single gene disorder caused by mutations in the SERPINA1 gene, which normally encodes a very abundan...
Editing of the AAT locus using novel base editing and prime editing technologies
PROJECT 2 - Project Summary/Abstract Gene editing has the potential to correct mutations and provide long-term therapeutic benefit for patients with rare monogenic diseases like...
Exploitation of the natural biodiversity of AAV to identify “super transducers
Project 3 - Project Summary: Liver is arguably the most permissive tissue for recombinant adeno-associated virus (rAAV)-mediated in vivo gene delivery, and liver transduction ca...
Modulation of Immune responses to gene therapy by Creation of AAV-specific Chimeric antigen receptor regulatory T cells (CAR-Tregs)
Project 4 - Abstract In Project 4, we will use our novel adeno-associated virus (AAV) directed chimeric antigen receptor (CAR) T-cells and T regulatory cells (Tregs) to modulate...
Models and Gene Therapies for AAT Deficiency
Administrative Core Project Summary The administrative core will provide a structure for management of the financial aspects of this complex multi-component program and create v...
Vector Immunology Core
CORE A - SUMMARY/ABSTRACT: The Vector Immunology Core will directly support all four projects of this program project grant by providing reliable, consistent and standardized im...
Human Genetic Disease Modeling and Physiology Core
Core B - Project Summary To have successful therapies for Alpha-1 antitrypsin deficiency it is necessary to have animal models that recapitulate the phenotypes see in human patie...
Optimized Gene Replacement for AAT deficiency and Modeling of Clinical Outcomes in small and large animal models
Project Summary (Project 1) Alpha-1 antitrypsin deficiency (AATD) is a common single gene disorder caused by mutations in the SERPINA1 gene, which normally encodes a very abundan...
Editing of the AAT locus using novel base editing and prime editing technologies
PROJECT 2 - Project Summary/Abstract Gene editing has the potential to correct mutations and provide long-term therapeutic benefit for patients with rare monogenic diseases like...
Exploitation of the natural biodiversity of AAV to identify “super transducers
Project 3 - Project Summary: Liver is arguably the most permissive tissue for recombinant adeno-associated virus (rAAV)-mediated in vivo gene delivery, and liver transduction ca...
Modulation of Immune responses to gene therapy by Creation of AAV-specific Chimeric antigen receptor regulatory T cells (CAR-Tregs)
Project 4 - Abstract In Project 4, we will use our novel adeno-associated virus (AAV) directed chimeric antigen receptor (CAR) T-cells and T regulatory cells (Tregs) to modulate...
Models and Gene Therapies for AAT Deficiency
Administrative Core Project Summary The administrative core will provide a structure for management of the financial aspects of this complex multi-component program and create v...
Vector Immunology Core
CORE A - SUMMARY/ABSTRACT: The Vector Immunology Core will directly support all four projects of this program project grant by providing reliable, consistent and standardized im...
Human Genetic Disease Modeling and Physiology Core
Core B - Project Summary To have successful therapies for Alpha-1 antitrypsin deficiency it is necessary to have animal models that recapitulate the phenotypes see in human patie...
Optimized Gene Replacement for AAT deficiency and Modeling of Clinical Outcomes in small and large animal models
Project Summary (Project 1) Alpha-1 antitrypsin deficiency (AATD) is a common single gene disorder caused by mutations in the SERPINA1 gene, which normally encodes a very abundan...
Editing of the AAT locus using novel base editing and prime editing technologies
PROJECT 2 - Project Summary/Abstract Gene editing has the potential to correct mutations and provide long-term therapeutic benefit for patients with rare monogenic diseases like...