Salivary Gland-Based Gene Therapy for Lysosomal Storage Diseases
基于唾液腺的溶酶体贮积病基因治疗
基本信息
- 批准号:7814758
- 负责人:
- 金额:$ 43.4万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2009
- 资助国家:美国
- 起止时间:2009-09-23 至 2012-11-30
- 项目状态:已结题
- 来源:
- 关键词:Adenovirus VectorAnimal ModelAnimalsBiodistributionBiomedical EngineeringBloodBlood CirculationBudgetsCellsCharacteristicsClimateClinical TrialsCollaborationsDNADataDependovirusDisadvantagedDoseEnzymesEvaluationFabry DiseaseFrequenciesGalactosidaseGasesGene DeliveryGene ExpressionGene TransferGenesGrantHandHumanHumoral ImmunitiesImmune responseInflammationInstitutionInterventionLaboratoriesLinkLysosomal Storage DiseasesMainstreamingMammalsMechanicsMethodsMicrobubblesMicrobubbles Ultrasound Contrast MediumMolecular AnalysisMouth DiseasesMusNon-Viral VectorOralOrganParentsPerformancePharmaceutical PreparationsPhasePhysiologicalPlasmaPlasmidsRelative (related person)ResearchResearch PersonnelRunningSafetySalivaSalivary GlandsSamplingSchoolsSystemSystemic diseaseTechnologyTestingTherapeuticTimeTissuesTitrationsTransgenesTranslationsTreatment ProtocolsUltrasonic TherapyUltrasonographyUniversitiesViralViral GenesViral VectorVisitWorkXerostomiaadenoviral-mediatedanalytical methodbaseexperiencegene therapygraduate studentindexingnon-viral gene deliveryparent projectplasmid DNApre-clinicalsonoporationuptakevectorviral gene delivery
项目摘要
DESCRIPTION (provided by applicant): This Competitive Revision Application for 4R00DE018188-02 proposes to expand the parent project to include a non-viral method of gene delivery based upon ultrasound destruction of DNA-associated microbubbles. This ultrasound-assisted gene transfer (UAGT) works differently from the viral-based methods of the parent project, but the end result, gene transfer to the salivary gland is the same.
Therefore, this supplement is proposed as a parallel but equivalent track, bringing in a co-investigator from within the Pi's affiliated institution who has almost twenty years of experience with ultrasoundmicrobubble technology. Figure 1 illustrates the relationship of the parent project and proposed supplement.
By executing the parent project and the proposed supplemental research plan in concert, we will be able to leverage the existing expertise acquired in the physiological and molecular analysis of salivary gland-based gene therapy for Fabry disease. This will create a climate where the translational potential of both viral and non-viral gene transfer methods can be objectively compared under tightly controlled conditions. With salivary gland-based gene therapy now in a Phase 1 human clinical trial, such comparisons are imperative if long-term gene expression in the salivary gland of humans is to be achieved.
The major mechanism for cementing collaboration between the Pl and the Co-l is the support of a floating graduate student who will participate in every step of the proposed supplemental project. This mechanism will link Dr. Wheatley's expertise in the construction and destruction of ultrasound
microbubble contrast agents with Dr. Passineau's gene therapy laboratory. The proposed budget includes allocation for this graduate student, based in the Drexel University School of Biomedical Engineering, to make frequent extended visits to Pittsburgh for animal work and sample analysis.
描述(由申请人提供):这份4R00DE018188-02的竞争性修订申请提议扩大母项目,包括一种基于超声破坏dna相关微泡的非病毒基因传递方法。这种超声波辅助基因转移(UAGT)的工作原理与母体项目中基于病毒的方法不同,但最终结果是,基因转移到唾液腺是相同的。
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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Michael J. Passineau其他文献
Michael J. Passineau的其他文献
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{{ truncateString('Michael J. Passineau', 18)}}的其他基金
Ultrasound-Assisted AQP1 Gene Therapy for Functional Restoration of Salivary Glan
超声辅助 AQP1 基因治疗唾液腺功能恢复
- 批准号:
8390748 - 财政年份:2012
- 资助金额:
$ 43.4万 - 项目类别:
Ultrasound-Assisted AQP1 Gene Therapy for Functional Restoration of Salivary Glan
超声辅助 AQP1 基因治疗唾液腺功能恢复
- 批准号:
8514570 - 财政年份:2012
- 资助金额:
$ 43.4万 - 项目类别:
Ultrasound-Assisted AQP1 Gene Therapy for Functional Restoration of Salivary Glan
超声辅助 AQP1 基因治疗唾液腺功能恢复
- 批准号:
8668774 - 财政年份:2012
- 资助金额:
$ 43.4万 - 项目类别:
Ultrasound-Assisted AQP1 Gene Therapy for Functional Restoration of Salivary Glan
超声辅助 AQP1 基因治疗唾液腺功能恢复
- 批准号:
8878038 - 财政年份:2012
- 资助金额:
$ 43.4万 - 项目类别:
Ultrasound-Assisted Gene Transfer of Anti-Biofilm Peptides to the Salivary Gland
超声辅助将抗生物膜肽基因转移至唾液腺
- 批准号:
8225139 - 财政年份:2011
- 资助金额:
$ 43.4万 - 项目类别:
Ultrasound-Assisted Gene Transfer of Anti-Biofilm Peptides to the Salivary Gland
超声辅助将抗生物膜肽基因转移至唾液腺
- 批准号:
8041732 - 财政年份:2011
- 资助金额:
$ 43.4万 - 项目类别:
Salivary Gland-Based Gene Therapy for Lysosomal Storage Diseases
基于唾液腺的溶酶体贮积病基因治疗
- 批准号:
7848134 - 财政年份:2008
- 资助金额:
$ 43.4万 - 项目类别:
Salivary Gland-Based Gene Therapy for Lysosomal Storage Diseases
基于唾液腺的溶酶体贮积病基因治疗
- 批准号:
7623847 - 财政年份:2008
- 资助金额:
$ 43.4万 - 项目类别:
Salivary Gland-Based Gene Therapy for Lysosomal Storage Diseases
基于唾液腺的溶酶体贮积病基因治疗
- 批准号:
7616605 - 财政年份:2008
- 资助金额:
$ 43.4万 - 项目类别:
Salivary gland-based gene therapy for lysosomal storage diseases
基于唾液腺的溶酶体贮积病基因治疗
- 批准号:
7244947 - 财政年份:2007
- 资助金额:
$ 43.4万 - 项目类别:
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