A Novel Vector Platform to Actualize T Cell Modification In Vivo

一种在体内实现 T 细胞修饰的新型载体平台

基本信息

  • 批准号:
    10663022
  • 负责人:
  • 金额:
    $ 42.76万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2023
  • 资助国家:
    美国
  • 起止时间:
    2023-08-04 至 2025-07-31
  • 项目状态:
    未结题

项目摘要

ABSTRACT To address the need for improved gene editing delivery systems, we propose constructing a chimeric “AdAAV” vector consisting of an adenovirus (Ad) with multiple adeno-associated viruses (AAVs) conjugated to its capsid's surface. We plan to employ the SpyTag/SpyCatcher technology to conjugate AAVs onto the Ad capsid. Of note in this regard, Ads are able to selectively target certain tissues (such as the liver in the case of huAd5) with high transduction efficiencies. Furthermore, our group has been able to engineer Ad fibers to facilitate tissue-specific targeting of T cells. In addition, by choosing an appropriate AAV serotype, and/or through capsid engineering AAVs can also selectively target desired tissues as well. AdAAVs may therefore provide superior targeting through the combined effects of both engineered Ad fiber and AAV capsid (which are matched to target the same tissue type). As a proof-of-concept, we plan to target T cells with AdAAVs. In addition, because AAVs carry single-stranded DNA, they can provide single-stranded donor templates, which are known to enhance the editing efficiency of homology directed repair (HDR). Within an AdAAV, the Cas protein can be encoded by the Ad genome while the single-stranded DNA template can be embodied within the genome of the AAVs. By virtue of the advantages of single-stranded donor templates for HDR, and by virtue of the high copy number of donor templates carried by the multiple AAVs associated with each Ad, this design might substantially increase editing efficiency. Finally, the AdAAV would possess a large packaging capacity since it would consist of a sum of the Ad's capacity (which is already high) and the AAV's capacity. Due to these factors, we suggest that AdAAVs may form a powerful and versatile new delivery system for gene editing therapies which overcomes many of the limitations associated with existing approaches. Our highly original AdAAV delivery system will greatly enhance the versatility of existing CRISPR-Cas gene editing therapies by circumventing several key obstacles to their broader applicability. The design features of AdAAV will potentially make it an ideal vector by which to address the challenges of gene editing delivery and thereby broadly enhance the general feasibility of gene editing-based therapies.
摘要

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)

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ monograph.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ sciAawards.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ conferencePapers.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ patent.updateTime }}

David Terry Curiel其他文献

David Terry Curiel的其他文献

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

{{ truncateString('David Terry Curiel', 18)}}的其他基金

Novel Vector Platform for Gene Therapy
用于基因治疗的新型载体平台
  • 批准号:
    10231536
  • 财政年份:
    2021
  • 资助金额:
    $ 42.76万
  • 项目类别:
Endothelial-targeted adenovirus for organ-selective gene editing in vivo
用于体内器官选择性基因编辑的内皮靶向腺病毒
  • 批准号:
    10228031
  • 财政年份:
    2019
  • 资助金额:
    $ 42.76万
  • 项目类别:
Novel Vector Platform for Gene Therapy
用于基因治疗的新型载体平台
  • 批准号:
    10388103
  • 财政年份:
    2019
  • 资助金额:
    $ 42.76万
  • 项目类别:
Endothelial-targeted adenovirus for organ-selective gene editing in vivo
用于体内器官选择性基因编辑的内皮靶向腺病毒
  • 批准号:
    9810634
  • 财政年份:
    2019
  • 资助金额:
    $ 42.76万
  • 项目类别:
In Vivo Editing for Hemophilia Gene Therapy
血友病基因治疗的体内编辑
  • 批准号:
    9695292
  • 财政年份:
    2018
  • 资助金额:
    $ 42.76万
  • 项目类别:
A 3D IN VITRO DISEASE MODEL OF ATRIAL CONDUCTION
心房传导 3D 体外疾病模型
  • 批准号:
    10166441
  • 财政年份:
    2017
  • 资助金额:
    $ 42.76万
  • 项目类别:
GORILLA ADENOVIRUS ZIKA VACCINE FOR HUMANS
人类大猩猩腺病毒寨卡疫苗
  • 批准号:
    9316943
  • 财政年份:
    2017
  • 资助金额:
    $ 42.76万
  • 项目类别:
A 3D IN VITRO DISEASE MODEL OF ATRIAL CONDUCTION
心房传导 3D 体外疾病模型
  • 批准号:
    10228624
  • 财政年份:
    2017
  • 资助金额:
    $ 42.76万
  • 项目类别:
Novel targeted adenovirus
新型靶向腺病毒
  • 批准号:
    9511780
  • 财政年份:
    2017
  • 资助金额:
    $ 42.76万
  • 项目类别:
Novel targeted adenovirus
新型靶向腺病毒
  • 批准号:
    10163752
  • 财政年份:
    2017
  • 资助金额:
    $ 42.76万
  • 项目类别:

相似海外基金

Rational design of rapidly translatable, highly antigenic and novel recombinant immunogens to address deficiencies of current snakebite treatments
合理设计可快速翻译、高抗原性和新型重组免疫原,以解决当前蛇咬伤治疗的缺陷
  • 批准号:
    MR/S03398X/2
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Fellowship
Re-thinking drug nanocrystals as highly loaded vectors to address key unmet therapeutic challenges
重新思考药物纳米晶体作为高负载载体以解决关键的未满足的治疗挑战
  • 批准号:
    EP/Y001486/1
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Research Grant
CAREER: FEAST (Food Ecosystems And circularity for Sustainable Transformation) framework to address Hidden Hunger
职业:FEAST(食品生态系统和可持续转型循环)框架解决隐性饥饿
  • 批准号:
    2338423
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Continuing Grant
Metrology to address ion suppression in multimodal mass spectrometry imaging with application in oncology
计量学解决多模态质谱成像中的离子抑制问题及其在肿瘤学中的应用
  • 批准号:
    MR/X03657X/1
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Fellowship
CRII: SHF: A Novel Address Translation Architecture for Virtualized Clouds
CRII:SHF:一种用于虚拟化云的新型地址转换架构
  • 批准号:
    2348066
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Standard Grant
BIORETS: Convergence Research Experiences for Teachers in Synthetic and Systems Biology to Address Challenges in Food, Health, Energy, and Environment
BIORETS:合成和系统生物学教师的融合研究经验,以应对食品、健康、能源和环境方面的挑战
  • 批准号:
    2341402
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Standard Grant
The Abundance Project: Enhancing Cultural & Green Inclusion in Social Prescribing in Southwest London to Address Ethnic Inequalities in Mental Health
丰富项目:增强文化
  • 批准号:
    AH/Z505481/1
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Research Grant
ERAMET - Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and paediatric medicines
ERAMET - 快速采用建模和模拟方法的生态系统,以满足孤儿药和儿科药物开发中的监管需求
  • 批准号:
    10107647
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    EU-Funded
Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and paediatric medicines
快速采用建模和模拟方法的生态系统,以满足孤儿药和儿科药物开发中的监管需求
  • 批准号:
    10106221
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    EU-Funded
Recite: Building Research by Communities to Address Inequities through Expression
背诵:社区开展研究,通过表达解决不平等问题
  • 批准号:
    AH/Z505341/1
  • 财政年份:
    2024
  • 资助金额:
    $ 42.76万
  • 项目类别:
    Research Grant
{{ showInfoDetail.title }}

作者:{{ showInfoDetail.author }}

知道了