HAIRPIN RIBOZYME GENE THERAPY FOR HBV INFECTION

发夹核酶基因疗法治疗乙型肝炎病毒感染

基本信息

  • 批准号:
    2535740
  • 负责人:
  • 金额:
    $ 37.49万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    1996
  • 资助国家:
    美国
  • 起止时间:
    1996-09-30 至 1999-08-31
  • 项目状态:
    已结题

项目摘要

Hepatitis B is a worldwide health problem. Our long term objective is to develop hairpin Ribozyme (Rz) gene therapy for the treatment of HBV infection. We have successfully completed both Specific Aims of our Phase I SBIR grant. In addition, we have modified two of the best ribozymes and evaluated their anti-viral effects in cells. The structurally modified ribozymes inhibited HBV production by at least 83%. These encouraging results indicate the feasibility of ribozyme-mediated gene therapy for the treatment of HBV infections. The work from the Phase I SBIR studies has been accepted for publication In GeNe Therapy (in press). Therefore, we further propose a Phase II SBIR study with the following aims: 1) to further optimize the two effective anti-HBV ribozymes (BR1-TL and BR3-TL) by varying the helix l length and kinetically characterizing the resulting variants to identify the ones with the greatest catalytic effects: 2) to generate viral vectors (adenoviral vectors and adeno-associated viral vectors) that express the single most active ribozyme genes identified and to generate vectors expressing both ribozymes in the same vector construct; and 3) to evaluate vector-mediated ribozyme activity in reducing HBV production in liver cell culture and evaluating pre-clinical efficacy and safety in a SCID-Hu mouse model. Achieving these Aims will generate the most effective ribozyme viral vector(s) to be used for a phase I clinical trial for treatment of HBV infection. PROPOSED COMMERCIAL APPLICATION: Immusol plans to commercially develop a Rz gene therapy approach to HBV infection. We will manufacture and provide Rz-expressing viral vectors in a vial. The vectors will then be provided to physicians around the world; they will carry out the in vivo intravenous vector-mediated gene transfer into patient liver cells to combat HBV virus.
乙型肝炎是一个全球性的健康问题。我们的长期目标是

项目成果

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JACK R BARBER其他文献

JACK R BARBER的其他文献

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{{ truncateString('JACK R BARBER', 18)}}的其他基金

RIBOZYMES FOR THE PREVENTION OF RESTENOSIS AFTER PTCA
用于预防 PTCA 术后再狭窄的核酶
  • 批准号:
    6184488
  • 财政年份:
    1998
  • 资助金额:
    $ 37.49万
  • 项目类别:
RIBOZYMES FOR THE PREVENTION OF RESTENOSIS AFTER PTCA
用于预防 PTCA 术后再狭窄的核酶
  • 批准号:
    6015667
  • 财政年份:
    1998
  • 资助金额:
    $ 37.49万
  • 项目类别:
RIBOZYMES FOR THE PREVENTION OF RESTENOSIS AFTER PTCA
用于预防 PTCA 术后再狭窄的核酶
  • 批准号:
    2678095
  • 财政年份:
    1998
  • 资助金额:
    $ 37.49万
  • 项目类别:
CHIMERIC ADENO/AVV VECTOR FOR GENE THERAPY AGAINST HCV
用于 HCV 基因治疗的嵌合腺苷/AVV 载体
  • 批准号:
    2643928
  • 财政年份:
    1998
  • 资助金额:
    $ 37.49万
  • 项目类别:
HAIRPIN RIBOZYME GENE THERAPY FOR HCV INFECTION
HCV 感染的发夹核酶基因疗法
  • 批准号:
    2865462
  • 财政年份:
    1997
  • 资助金额:
    $ 37.49万
  • 项目类别:
HAIRPIN RIBOZYME GENE THERAPY FOR HCV INFECTION
HCV 感染的发夹核酶基因疗法
  • 批准号:
    2536407
  • 财政年份:
    1997
  • 资助金额:
    $ 37.49万
  • 项目类别:
HAIRPIN RIBOZYME GENE THERAPY FOR HCV INFECTION
HCV 感染的发夹核酶基因疗法
  • 批准号:
    6170744
  • 财政年份:
    1997
  • 资助金额:
    $ 37.49万
  • 项目类别:
RAPID GENERATION OF RAAV PACKAGING CELL LINES USING FACS
使用 FACS 快速生成 RAAV 包装细胞系
  • 批准号:
    2422987
  • 财政年份:
    1997
  • 资助金额:
    $ 37.49万
  • 项目类别:
HAIRPIN RIBOZYME GENE THERAPY FOR HBV INFECTION
发夹核酶基因疗法治疗乙型肝炎病毒感染
  • 批准号:
    2672687
  • 财政年份:
    1996
  • 资助金额:
    $ 37.49万
  • 项目类别:

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