Abstract

Many of the hereditary diseases are related to defects in genes that would otherwise be expressed in hepatocytes. Examples of these diseases including alpha-1-antitrypsin deficiency and hemophilia. The development of an efficient gene therapy vector to target genes into hepatocytes of patients with these types of hereditary diseases can potentially improve the quality of lives of these patients. Hepatitis B virus (HBV) is a DNA virus with a high liver-specificity. A gene-therapy vector based on this virus will likely be an effective vector for targeting genes to hepatocytes. Furthermore, unlike current gene-therapy protocols which often involve complicated surgical procedures, patients can be treated with simple i.v. injections of the recombinant HBV virus carrying the desired gene. The goal of this project is to develop an HBV-based vector for liver-targeted gene therapy.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Center Core Grants (P30)
Project #
5P30DK048522-03
Application #
6239169
Study Section
Project Start
1997-03-01
Project End
1998-02-28
Budget Start
1996-10-01
Budget End
1997-09-30
Support Year
3
Fiscal Year
1997
Total Cost
Indirect Cost

  Institution

Name
University of Southern California
Department
Type
DUNS #
041544081
City
Los Angeles
State
CA
Country
United States
Zip Code
90089

  Publications

Ju, Yaping; Janga, Srikanth Reddy; Klinngam, Wannita et al. (2018) NOD and NOR mice exhibit comparable development of lacrimal gland secretory dysfunction but NOD mice have more severe autoimmune dacryoadenitis. Exp Eye Res 176:243-251
Peddi, Santosh; Pan, Xiaoli; MacKay, John Andrew (2018) Intracellular Delivery of Rapamycin From FKBP Elastin-Like Polypeptides Is Consistent With Macropinocytosis. Front Pharmacol 9:1184
Zhou, Beiyun; Flodby, Per; Luo, Jiao et al. (2018) Claudin-18-mediated YAP activity regulates lung stem and progenitor cell homeostasis and tumorigenesis. J Clin Invest 128:970-984
Khanova, Elena; Wu, Raymond; Wang, Wen et al. (2018) Pyroptosis by caspase11/4-gasdermin-D pathway in alcoholic hepatitis in mice and patients. Hepatology 67:1737-1753
Zhang, Chunying; Niu, Chao; Yang, Kevin et al. (2018) Human esophageal myofibroblast secretion of bone morphogenetic proteins and GREMLIN1 and paracrine regulation of squamous epithelial growth. Sci Rep 8:12354
Tsai, Yuan-Li; Ha, Dat P; Zhao, He et al. (2018) Endoplasmic reticulum stress activates SRC, relocating chaperones to the cell surface where GRP78/CD109 blocks TGF-? signaling. Proc Natl Acad Sci U S A 115:E4245-E4254
Chen, Jingwen; Lam, Albert T; Zhang, Yong (2018) A macrodomain-linked immunosorbent assay (MLISA) for mono-ADP-ribosyltransferases. Anal Biochem 543:132-139
Chang, Huiyi H; Yeh, Jih-Chao; Ichiyama, Ronaldo M et al. (2018) Mapping and neuromodulation of lower urinary tract function using spinal cord stimulation in female rats. Exp Neurol 305:26-32
Chen, Chien-Yu; Chen, Jingyu; He, Lina et al. (2018) PTEN: Tumor Suppressor and Metabolic Regulator. Front Endocrinol (Lausanne) 9:338
Nakamura, Brooke N; Glazier, Alison; Kattah, Michael G et al. (2018) A20 regulates canonical wnt-signaling through an interaction with RIPK4. PLoS One 13:e0195893

Showing the most recent 10 out of 449 publications