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Grant Funding

The Board of Governors Gene Therapeutics Research Institute (GTRI) receives funding via grants, which support several programs in a variety of research areas. If the programmatic nature of the work is within the scope of GTRI-managed program project and center grants, the investigator will be eligible for supported services in accordance with sponsor and Principal Investigator guidelines. The following is a list of current and past GTRI grants, which provide support to its research programs.

National Institutes of Health # R01 NS06117
Brain immune responses: Cellular & Molecular Mechanisms Major Goals: To explore the cellular and molecular basis of glial cell responses to immune attack both in vivo and in vitro.
Dr. P. R. Lowenstein (PI)

National Institutes of Health # R01 NS057711
Engineering the Brain Immune System for Tumor Therapy
Major Goal: Recruit dendritic cells and macrophages into the glioma mass in situ to mount a specific immune response against the tumor: efficacy of gene therapy vs. DC and tumor antigen vaccination approaches.
Dr. Maria G Castro (PI)

National Institutes of Health # R21 NS054143
Targeted Toxin for Brain Cancer Gene Therapy
Major Goals: Develop a targeted glioma gene therapy approach with high therapeutic efficacy and negligible toxicity which could be translated into clinical trials
Dr. Maria G Castro (PI)

National Institutes of Health # R01 NS054193
Gene Therapy and the Brain: Neuroimmune Interactions
Major Goal: To develop techniques to modulate immune regulatory mechanisms to lead to safer and more effective clinical gene therapy trials designed to treat neurological pathologies: applications to brain cancer.
Dr. P. R. Lowenstein (PI)

National Institutes of Health # U01 NS052465
Gutless Adenovirus Mediated Gene Therapy for Glioma
Major Goals: Assess efficacy, bio-distribution and toxicology of high capacity adenovirus vectors encoding HSV1-TK and Flt3L to be used for the treatment of glioblastoma in a phase1/2 clinical trial. Submission of IND.
Dr. Maria G. Castro (PI: Dr. P. R. Lowenstein)

National Institutes of Health # R01 NS061107
Brain immune responses: Cellular & Molecular Mechanisms
Brain immune responses: Cellular & Molecular Mechanisms.
Major Goals: Cellular and molecular basis of glial cell responses to immune attack both in vivo and in vitro: implications for uncovering novel therapeutic target for glioblastoma multiforme.
Dr. Maria G. Castro (PI: Dr. P. R. Lowenstein)

National Institutes of Health # F32 NS058156
Gutless Adenovirus Mediated Targeted Therapeutics for Glioma
Major goal: Targeted glioma gene therapy approach that elicits high efficacy and negligible toxicity.
Dr. Maria G Castro Sponsor (PI: M. Candolfi)

National Institutes of Health/#1 R01 NS44556.01
Gene Therapy for Chronic Neurodegenerative Disorders
National Institutes of Health/#1 R01 NS44556.01
Gene Therapy for Chronic Neurodegenerative Disorders
Major Goals: We will test the hypothesis that long term expression of Glial Derived Neurotrophic Factor (GDNF) from a gutless adenovirus vector and under the control of an inducible promoter, will be stable, non-toxic, non-immunogenic and it will not elicit adverse behavioural or neuroanatomical side effects.
Dr. M.G. Castro: Principal Investigator
Dr. P.R. Lowenstein: Co-Principal Investigator

National Institutes of Health/#1 R01 NS42893 01
Gene Therapy for Neurological Diseases
Major Goals: To develop novel high-capacity adenoviral systems for long term, stable, non-cytotoxic, and non-immunogenic delivery of neuro-protective genes to the brain for both experimental transgene expression in adult animals, and for the future treatment of chronic neurodegenerative diseases such as PD and Alzheimers's disease by gene therapy.
Dr. P.R. Lowenstein: Principal Investigator
Dr. M.G. Castro: Co-Principal Investigator

Linda Tallen & David Paul Kane Foundation
Annual Fellow in cancer gene therapy
Gene Therapy for Cancer Major goals: To develop novel gene therapies to treat brain cancers using immune modulatory genes.
Drs. M.G. Castro, P.R. Lowenstein: Principal Investigators

Jonsson Comprehensive Cancer Center/UCLA
Brain Tumor Stem Cells
Major goals: Cancer stem cells are self-renewing, multipotent, and capable, on a clonal level, of producing an entire, heterogeneous tumor mass. It has been theorized that curative cancer therapies will need to specifically attack these cancer stem cells. The current proposal examines the relationship between brain tumor progenitors and neural stem cells and is designed test the hypotheses that brain tumors contain cancer stem cells and that therapies directed towards blocking cancer stem cell self renewal will be effective treatments in animal models.
Drs. M.G. Castro, P.R. Lowenstein: Co-Investigators

National Institute of Health #: 1 RO3 TW006273-01A1
Apoptosis of Anterior Pituitary Cells
Major Goals: To assess the molecular pathways that mediates all death within anterior pituitary gland. These molecular targets might provide novel approaches for developing gene therapies to treat pituitary tumors.
Dr. M.G. Castro: Principal Investigador

National Institutes of Health #1 R21 NS047298-01
Gene Therapy for Glioma
Major Goals: To develop a novel gene therapy approach based on high capacity adenovirus vectors expressing cytotoxic genes for the treatment of glioblastoma.
Dr. P. R. Lowenstein - Principal Investigator
Dr. M. G. Castro: Co-Principal Investigator

National Institutes of Health #1 RO1 NS048959-01
Bone Marrow Derived Neural Stem Cell Therapy for Glioma
Major Goals: To develop stem cell therapy for the treatment of glioblastoma multiforme
Dr. John Yu, Neurological Institute, CSMC - Principal Investigator)
Drs. Maria G. Castro, P. R. Lowenstein - Co-Investigators

National Institutes of Health T32 Training Grant/UCLA
Interdisciplinary training in Virology and Gene Therapy
Major Goals: Developments in virology have led to the advancement of several disciplines, including gene therapy. The key issue in gene therapy is the development of delivery vehicles. To fulfil the national need, the UCLA Virology and Gene Training Program aims to provide a unique and outstanding environment for pre-doctoral students pursuing research careers in fields related to gene therapy. At UCLA, this rigorous training program with emphasis on vertical integration of basic sciences and therapeutic applications will produce scientists required for long-term development of gene therapy.
Dr. Maria Castro, Dr. P.R. Lowenstein: Mentors

National Institutes of Health T32 Training Grant
Training Program in Endocrinology and Diabetes
Major Goals: To provide a unique and outstanding environment for post-doctoral students pursuing research careers in fields related to endocrinology, neuro-endocrinology and metabolism at Cedars Sinai Medical Center/UCLA. This rigorous training program with emphasis on vertical integration of basic sciences and therapeutic applications will produce scientists required for long-term development of the endocrinology and diabetes field.
Dr. Shlomo Melmed - Principal Investigator
Dr. Maria Castro, Dr. P.R. Lowenstein: Mentors

National Institutes of Health #2 R01 EY13431
Mechanisms of Epithelial Alterations in Diabetic Cornea
Major Goals: To facilitate the development of gene-based therapies for diabetic corneal abnormalities
Dr. Maria G. Castro: Co-Investigator (Dr. A. Ljubimov - Principal Investigator)

National Institutes of Health #3 R01 NS044556-03S1
Gene therapy for chronic neurodegenerative disorders (Minority research supplement)
Major Goals: To test the hypothesis that the presence of an immune response to the transactivator will not affect the regulated expression of transgene in the central nervous system (CNS)
Dr. Maria G. Castro: Principal Investigator

National Institutes of Health # 1 F32 NS053034-01
Glioma regression using gutless adenoviral vectors
Major Goals: To test the hypothesis that use of high capacity, gutless adenoviral vectors will allow tumor regression and prolong survival even in the presence of pre-existing immunity to adenovirus.
Dr. G. King - Principal Investigator
Dr. Maria G. Castro - Sponsor and Mentor

#5R01 NS042893-03
National Institutes of Health
Gene Therapy for neurological diseases
(Minority research supplement)
Major goal: To develop in vivo imaging techniques to assess therapeutic efficacy in pre-clinical models of neurodegeneration. Clinical platforms for imaging of PD patients.
Dr. P.R. Lowenstein, Principal Investigator

Multiple Sclerosis Society of Canada Major goals: Beside the loss of oligodendricytes and myelin, there is now extensive evidence that axonal and neuronal destruction is a common pathological feature in neuroinflammatory diseases such as Multiple Sclerosis and may account for the progressive disability that occurs in the disease. By creating neuron specific transgenic mice, we will be able to study the immune response against neurons as well as for the glial cells in the central and peripheral nervous systems. Gene therapy vectors will be used for neuronal targeting for neuromodulation and therapy. This study provides novel avenues to regulate neuroimmune interactions in brain diseases such as multiple sclerosis, or during the implementation of novel gene therapies in the brain.
Daniel Larocque, Principal Investigator
Dr. P.R. Lowenstein, Dr. Maria G. Castro, Sponsors and Mentors

Human Frontier Science Program
Immune mediated gene expression in neurons
Major goals: Beside the loss of oligodendricytes and myelin, there is now extensive evidence that axonal and neuronal destruction is a common pathological feature in neuroinflammatory diseases such as Multiple Sclerosis and may account for the progressive disability that occurs in the disease. By creating neuron specific transgenic mice, we will be able to study the immune response against neurons as well as for the glial cells in the central and peripheral nervous systems. Gene therapy vectors will be used for neuronal targeting for neuromodulation and therapy. This study provides novel avenues to regulate neuroimmune interactions in brain diseases such as multiple sclerosis, or during the implementation of novel gene therapies in the brain.
Daniel Larocque, Principal Investigator
Dr. P.R. Lowenstein, Dr. Maria G. Castro, Sponsors and Mentors

National Institutes of Health/ #U54 4NS 04-5309
Parkinson's Disease Study Group
Interorganizational study of Parkinson's Disease under a consortium agreement.
Major Goals: The ultimate goal of this proposal is to develop novel high-capacity adenoviral systems for long-term, stable, non-cytotoxic, and non-immunogenic delivery of neuroprotective genes to the brain for the future treatment of chronic neurodegenerative disease such as Parkinson¿s disease by gene therapy.
Co-Principal Investigators (Cedars-Sinai Medical Center project)

U.S. Department of Energy DE-FG02-04ER63830
Gene Therapeutics Research Program
Major Goal: Develop the infrastructure for in-vivo preclinical gene therapy protocols as they apply to brain cancer, neurodegenerative diseases and endocrine disorders
Dr. P.R. Lowenstein - Principal Investigator
Dr. Maria G. Castro - Co-Investigator

European Community Framework V Annual Funding: Reagents and technology transfer
Targeted vectors for cancer gene therapy: receptor and transcriptional targeting.
Major Goals: Development and testing of new vector systems which will enable specific tissue targeting therefore enhancing their efficiency and down modulating putative toxic side effects. The overall aim is to develop vectors, which could be delivered systemically for tumor treatment.
Dr. P. R. Lowenstein, Co-investigator