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High-probability grants
According to our matching algorithm, Louise R. Rodino-Klapac is the likely recipient of the following grants.
Years |
Recipients |
Code |
Title / Keywords |
Matching score |
2007 — 2009 |
Rodino-Klapac, Louise R. |
F32Activity Code Description: To provide postdoctoral research training to individuals to broaden their scientific background and extend their potential for research in specified health-related areas. |
Dystrophin Restoration in Two Animal Models of Duchenne Muscular Dystrophy @ Research Inst Nationwide Children's Hosp
[unreadable] DESCRIPTION (provided by applicant): We are developing a gene based therapeutic for boys with Duchenne muscular dystrophy (DMD). DMD only affects boys since it is on the X-chromosome which determines the sex of the baby. It causes muscle weakness due to a defective gene that results in the loss of an important muscle protein called Dystrophin. Presently there is no effective cure for DMD and only limited treatment options. Prednisone, a steroid with potentially serious side effects, provides partial benefit, but it does have significant side-effects after longterm use. To develop a cure, we are attempting to manipulate and replace the defective gene into two animal models of DMD with a smaller but functional dystrophin gene (micro-dystrophin). The microdystrophin gene will be transferred into muscle by adeno-associated virus (AAV). This virus does not cause any known human disease, making it appropriate for safe use in a clinical trial. The premise for this gene therapy proposal is that clinically meaningful results can be achieved by vascular delivery in patients with Duchenne muscular dystrophy (DMD). Furthermore, we think it is likely that FDA will move in a stepwise fashion toward vascular delivery whereby regional vascular delivery will precede systemic vascular delivery for clinical trials. This proposal revolves around this central point and gene delivery to the legs through the femoral artery is the starting point. The first goal for this project (Aim 1) is to define the best adeno-associated virus (AAV) serotype to cross the vascular barrier when delivered through the femoral artery of the mdx mouse. We will first define the best AAV serotype that crosses the vascular barrier carrying micro-dystrophin driven by a truncated muscle creatine kinase (MCK) promoter delivered through the femoral artery of the mdx mouse. The dog environment for gene transfer more closely matches clinical challenges because of size and immunogenicity related to outbreeding. Success in the dog will establish dosing and delivery parameters more closely simulating clinical trials and define guidelines for immunosuppression. Next, we will define the immunosuppression regimen required in the dog model using wild-type dogs to develop assays, and then the GRMD model to define the immunosuppression reagent ideal for expression of micro-dystrophin (Aim 2). Finally, the optimal AAV serotype carrying micro-dystrophin in the mouse will be perfused into the femoral artery of the golden retriever muscular dystrophy (GRMD) dog (Aim 3). [unreadable] [unreadable] [unreadable]
|
0.915 |
2016 — 2020 |
Rodino-Klapac, Louise R. |
P50Activity Code Description: To support any part of the full range of research and development from very basic to clinical; may involve ancillary supportive activities such as protracted patient care necessary to the primary research or R&D effort. The spectrum of activities comprises a multidisciplinary attack on a specific disease entity or biomedical problem area. These grants differ from program project grants in that they are usually developed in response to an announcement of the programmatic needs of an Institute or Division and subsequently receive continuous attention from its staff. Centers may also serve as regional or national resources for special research purposes. |
Core 2: Therapeutic Viral Vector Design and Development Core @ Research Inst Nationwide Children's Hosp
Project Summary - Core 2 The goal of the Therapeutic Viral Vector Design and Development Core (Core2) is to guide viral vector design, produce high quality viral vectors, and support Investigator initiated product development for novel vector based biologics with the goal of moving them to an IND. Significantly, the services of the Vector D & D Core represent a fundamental necessity to the defined and developmental projects within this CORT application as well as the larger identified Research Base. The center now holds 6 INDs for AAV gene therapy in various muscular dystrophies. We have gained valuable experience through our interactions with the FDA regarding the preclinical and toxicology/biodistribution studies required for IND approval. As part of this Core, Dr. Rodino-Klapac and her team will provide guidance for each project on the proper design of viral vectors and implementation of IND enabling preclinical studies. Understanding the translational pathway facilitates coordination between the PI's research team and the Vector Core for the production and testing of rAAV and other viral vectors in a manner consistent with FDA expectations at all phases of product development. This translates into a uniform vector product as a result of using consistent processes from early pre-clinical preps, through tox vector generation and ultimately early phase clinical grade vector produced using phase appropriate cGMPs. Through the demonstrated support of the Research Base within the Center for Gene Therapy at NCH and outside external collaborations, the NCH Vector Core has garnered such experience. The infrastructure support supplied by this P50 mechanism will allow for production of pre-clinical grade vectors for the three projects as well as guidance on vector design and preclinical implementation for each project in addition to the defined Research Base. Moreover, continued refinement of critical production processes and test methods as well as feasibility testing of novel AAV vector modalities will provide greater value to research base Investigators by reducing cost and time to completion with better quality and enhanced capacity. Our Specific Aims build upon the existing Vector Core infrastructure at NCH and will fulfill the primary goal of this CORT application - to accelerate the translation of basic science projects into genetic therapies for muscular dystrophies. In summary, direct interaction between the Core and Investigative team from each of the three projects will leverage product and regulatory experience to accelerate timelines for defined products and provide guidance where it was lacking to enable new laboratories to move proof of concept studies rapidly into translational research. The ultimate effect of this aligns perfectly with P50 CORT program mission, which includes the development of ?tangible products or deliverables? for the ?development of more effective treatments? for human disease in which case we will putting more novel biologics into the clinical trial pipeline for treatment of a host of musculoskeletal diseases.
|
0.915 |