Research Topics
Species | Sihong SongSummaryAffiliation: University of Florida Country: USA Publications
Research Grants
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Detail Information
Publications
Alpha1-antitrypsin gene therapy modulates cellular immunity and efficiently prevents type 1 diabetes in nonobese diabetic miceYuanqing Lu
Department of Pharmaceutics, University of Florida, Gainesville, FL 32610, USA
Hum Gene Ther 17:625-34. 2006..These results strongly suggest that rAAV1-mediated AAT gene therapy may be useful as a novel approach to prevent type 1 diabetes...
The protective effects of plasma gelsolin on stroke outcome in ratsHuong T Le
Department of Pharmaceutics, University of Florida College of Pharmacy, Gainesville, FL 32610, USA
Exp Transl Stroke Med 3:13. 2011..abstract:..
Ex vivo transduced liver progenitor cells as a platform for gene therapy in miceSihong Song
Department of Pharmaceutics, University of Florida, Gainesville, FL 32610, USA
Hepatology 40:918-24. 2004....
DNA-dependent PK inhibits adeno-associated virus DNA integrationSihong Song
Department of Pharmaceutics, Powell Gene Therapy Center, Genetics Institute, University of Florida, Gainesville, FL 32610, USA
Proc Natl Acad Sci U S A 101:2112-6. 2004..In summary, we have provided evidence that DNA-PK inhibits AAV integration both in vitro and in vivo...
Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vectorYuanqing Lu
Department of Pharmaceutics, University of Florida College of Pharmacy, Gainesville, FL 32610, USA
J Gene Med 8:730-5. 2006..These results provide strong support for the functionality of AAT in ongoing clinical studies of muscle-directed AAT gene therapy...
Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vectorThomas J Conlon
Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32608, USA
Mol Ther 12:867-75. 2005..Therefore, pseudotyped AAV8 provides a vehicle to infect a high percentage of hepatocytes stably and thereby express therapeutic molecules to modify AAT PiZ transcripts...
Ex vivo transduction and transplantation of bone marrow cells for liver gene delivery of alpha1-antitrypsinHong Li
Department of Pharmaceutics, University of Florida, Gainesville, FL, USA
Mol Ther 18:1553-8. 2010..These results demonstrated that rAAV vector-mediated BM cell-based liver gene therapy is feasible for the treatment of AAT deficiency and implies a novel therapy for the treatment of liver diseases...
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adultsMark L Brantly
Department of Medicine, University of Florida, Gainesville, FL 32611, USA
Hum Gene Ther 17:1177-86. 2006....
Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse modelChristian Grimstein
Department of Pharmaceutics, University of Florida, Gainesville, FL 32610, USA
J Transl Med 9:21. 2011..In the present study we investigated the feasibility of hAAT monotherapy for the treatment of chronic arthritis in collagen-induced arthritis (CIA), a mouse model of rheumatoid arthritis (RA)...
Adipose tissue-derived mesenchymal stem cell-based liver gene deliveryHong Li
Departments of Pharmaceutics, University of Florida, Gainesville, FL, USA
J Hepatol 54:930-8. 2011..In the present study, we investigated the feasibility of AT-MSC-based liver gene delivery for the treatment of alpha 1-antitrypsin deficiency...
Alpha1-antitrypsin protects beta-cells from apoptosisBin Zhang
Department of Pharmaceutics, University of Florida, Gainsville, FL 32610, USA
Diabetes 56:1316-23. 2007..These results propose a novel biological function for this molecule and suggest it may represent an effective candidate for attempts seeking to prevent or reverse type 1 diabetes...
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectorsYuanqing Lu
Department of Pharmaceutics, Powell Gene Therapy Center, Genetics Institute, University of Florida, Gainesville, FL 32610, USA
Proc Natl Acad Sci U S A 106:17158-62. 2009..These results demonstrate the immunogenic differences of rAAV1 and rAAV8 and imply tremendous potential for these vectors in different applications, where an immune response to transgene is to be either elicited or avoided...
Systemic overexpression of IL-10 induces CD4+CD25+ cell populations in vivo and ameliorates type 1 diabetes in nonobese diabetic mice in a dose-dependent fashionKevin S Goudy
Department of Pathology, Powell Gene Therapy Center, University of Florida, 1600 SW Archer Road, Gainesville, FL 32610, USA
J Immunol 171:2270-8. 2003..This study indicates the potential for immunomodulatory gene therapy to prevent autoimmune diseases, including type 1 diabetes, and implicates IL-10 as a molecule capable of increasing the percentages of regulatory cells in vivo...
Glucose transporter-2 (GLUT2) promoter mediated transgenic insulin production reduces hyperglycemia in diabetic miceBrant R Burkhardt
Department of Pathology, University of Florida College of Medicine, Gainesville, 32610, USA
FEBS Lett 579:5759-64. 2005..These findings indicate that the GLUT2 promoter may be a potential candidate for regulating transgenic insulin production for hepatic insulin gene therapy in the treatment of type I diabetes...
Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspectsSihong Song
Powell Gene Therapy Center of the University of Florida Genetics Institute, University of Florida, Gainesville, Florida 32615, USA
Mol Ther 6:329-35. 2002..These studies indicate that the risks of immune reaction and germline transmission after intramuscular injection of rAAV-SERPINA1 in nonhuman primates are relatively low within the range of vector doses studied...
Combination of alpha-1 antitrypsin and doxycycline suppresses collagen-induced arthritisChristian Grimstein
Department of Pharmaceutics, University of Florida, Gainesville, FL 32610, USA
J Gene Med 12:35-44. 2010..In order to develop a new treatment option, the present study employs a gene therapy-based combination therapy using doxycycline and human alpha-1 antitrypsin (hAAT)...
The effect of DNA-dependent protein kinase on adeno-associated virus replicationYoung Kook Choi
Department of Pharmaceutics, University of Florida, Gainesville, Florida, United States of America
PLoS ONE 5:e15073. 2010..DNA-dependent protein kinase (DNA-PK) is a DNA repair enzyme and plays an important role in determining the molecular fate of the rAAV genome. However, the effect this cellular enzyme on rAAV DNA replication remains elusive...
Immune depletion with cellular mobilization imparts immunoregulation and reverses autoimmune diabetes in nonobese diabetic miceMatthew J Parker
Department of Pathology, University of Florida, Gainesville, Florida, USA
Diabetes 58:2277-84. 2009....
Improved method of recombinant AAV2 delivery for systemic targeted gene therapyCathryn Mah
Department of Pediatrics, University of Florida, Gainesville, Florida 32610, USA
Mol Ther 6:106-12. 2002..These studies demonstrate a novel method to deliver rAAV vectors more effectively that could prove to be a successful alternative mode of virus-mediated human gene therapy...
A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysemaIrina Petrache
Division of Pulmonary and Critical Care, Department of Medicine, John Hopkins University School of Medicine, Baltimore, Maryland, USA
Am J Respir Crit Care Med 173:1222-8. 2006..The association of alpha1-antitrypsin deficiency with the development of emphysema has supported the concept that protease/antiprotease imbalance mediates cigarette smoke-induced emphysema...
Enhancing rAAV vector expression in the lungIsabel Virella-Lowell
Department of Pediatrics of the University of Florida College of Medicine, USA
J Gene Med 7:842-50. 2005..Although the advantage of rAAV5 over rAAV2 in the lung has already been described, the availability of another serotype (rAAV1) capable of efficient gene transfer in the lung could be useful...
Research Grants
- Anti-inflammatory Serpin(AAT and Elafin) Gene Transfers*Sihong Song; Fiscal Year: 2003..abstract_text> ..
- Gene Therapy for Correction of PiZ MutationSihong Song; Fiscal Year: 2006..Liver pathology will be examined. We anticipate achieving the therapeutic correction in PiZ transgenic mouse model. The success of this project may lead to a novel gene therapy for clinical treatment of human AAT deficiency. ..
