RICHARD J contact SAMULSKI

Summary

Affiliation: University of North Carolina
Country: USA

Publications

  1. ncbi request reprint AAV's anatomy: roadmap for optimizing vectors for translational success
    Angela M Mitchell
    UNC Gene Therapy Center, University of North Carolina at Chapel Hill, 7119 Thurston Bowles Building, 104 Manning Drive, Campus Box 7352, Chapel Hill, NC 27599 7352, USA
    Curr Gene Ther 10:319-40. 2010
  2. ncbi request reprint Biosafety of Recombinant Adeno-associated Virus Vectors
    David J Dismuke
    UNC Gene Therapy Center, University of North Carolina at Chapel Hill, 7119 Thurston Bowles Building, 104 Manning Drive Campus Box 7352, Chapel Hill, NC 27599 7352, USA
    Curr Gene Ther 13:434-52. 2013
  3. pmc AAV exploits subcellular stress associated with inflammation, endoplasmic reticulum expansion, and misfolded proteins in models of cystic fibrosis
    Jarrod S Johnson
    Department of Pharmacology, University of North Carolina, Chapel Hill, North Carolina, United States of America
    PLoS Pathog 7:e1002053. 2011
  4. pmc Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles
    Wuping Li
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 16:1252-60. 2008
  5. doi request reprint Efficient retrograde neuronal transduction utilizing self-complementary AAV1
    Edmund R Hollis
    Department of Neurosciences, University of California, San Diego, La Jolla, California 92093, USA
    Mol Ther 16:296-301. 2008
  6. ncbi request reprint Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose
    Zhijian Wu
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 16:280-9. 2008
  7. ncbi request reprint Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications
    Joshua C Grieger
    University of North Carolina Gene Therapy Center, Chapel Hill 27599, USA
    Adv Biochem Eng Biotechnol 99:119-45. 2005
  8. ncbi request reprint Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells
    TERESA BORRAS
    Department of Ophthalmology, University of North Carolina School of Medicine, 103 Mason Farm Road, Chapel Hill, NC 27599, USA
    J Gene Med 8:589-602. 2006
  9. ncbi request reprint Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency
    Ulrich T Hacker
    Klinik fur Innere Medizin I, Klinikum der Universität zu Köln, Joseph Stelzmann Strasse 9, 50925 Köln, Germany
    J Gene Med 7:1429-38. 2005
  10. pmc Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors
    Sifeng Chen
    Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA
    Hum Gene Ther 16:235-47. 2005

Research Grants

  1. AAV Gene Therapy for AAT deficiency
    RICHARD SAMULSKI; Fiscal Year: 2009
  2. Rational and combinatorial engineering of AAV vectors
    Richard J Samulski; Fiscal Year: 2010
  3. CTL response to AAV Vector
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
  4. Differential regulation of multiple transgenes for treatment of eye disease
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
  5. AAV Gene Therapy for AAT deficiency
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
  6. CTL response to AAV Vector
    Chengwen Li; Fiscal Year: 2011
  7. Rational and combinatorial engineering of AAV vectors
    Richard J Samulski; Fiscal Year: 2011
  8. MUSCLE DELIVERY OF AAV VECTORS FOR GENE THERAPY
    RICHARD SAMULSKI; Fiscal Year: 2001
  9. rAAV Revitalized Structural Allografts
    RICHARD SAMULSKI; Fiscal Year: 2005
  10. Muscle-specific AAV Vectors for DMD Gene Therapy
    RICHARD SAMULSKI; Fiscal Year: 2007

Collaborators

  • Tal Kafri
  • Liqun Zhang
  • Raymond J Pickles
  • Martina Gentzsch
  • Joshua C Grieger
  • Haiyan Fu
  • Terence R Flotte
  • Luca Perabo
  • S Zolotukhin
  • Thomas McCown
  • Mark Tuszynski
  • P E Monahan
  • Douglas M McCarty
  • Hildegard Buning
  • Chengwen Li
  • Jarrod S Johnson
  • Zhijian Wu
  • Aravind Asokan
  • David J Dismuke
  • Angela M Mitchell
  • Wuping Li
  • Edmund R Hollis
  • Terry van Dyke
  • Mavis Agbandje-McKenna
  • TERESA BORRAS
  • Sifeng Chen
  • Arvind Asokan
  • Ulrich T Hacker
  • Pierre Chenuaud
  • Liliane Tenenbaum
  • Boris Kantor
  • Carla M P Ribeiro
  • Sarah C Nicolson
  • Jayme K Warischalk
  • Ken Kadoya
  • Taiping Zhang
  • Stefan Leichtle
  • Nina DiPrimio
  • D Eugene Redmond
  • Matthew Hirsch
  • Chaoying Yin
  • Junjiang Sun
  • Lakshmanan Govindaswamy
  • Fang Yin
  • Norman E Sharpless
  • Kimberly B Petermann
  • Julie B Hamra
  • Jeffrey S Bartlett
  • Guorong Li
  • Lakshmanan Govindasamy
  • Vivian W Choi
  • Sarah S Chisolm
  • Wei Xue
  • Martha Campbell-Thompson
  • Sandra Lutz
  • Michael Hallek
  • William W Hauswirth
  • Susan B S King
  • Kirsten M Madsen
  • Olena Y Glushakova
  • Tobias Herold
  • Scott A Loiler
  • Lisa Wingenfeld
  • David M Kofler
  • Kenneth I Berns
  • Mark A Atkinson
  • Natascha K Schuhmann
  • C Craig Tisher
  • Anupam Agarwal
  • Franz M Gerner
  • Matthias Kapturczak
  • Joseph Rabinowitz
  • Joseph E Rabinowitz
  • Nathalie Provost
  • Philippe Moullier
  • Thibaut Larcher
  • Yan Cherel
  • Nicole Casadevall

Detail Information

Publications17

  1. ncbi request reprint AAV's anatomy: roadmap for optimizing vectors for translational success
    Angela M Mitchell
    UNC Gene Therapy Center, University of North Carolina at Chapel Hill, 7119 Thurston Bowles Building, 104 Manning Drive, Campus Box 7352, Chapel Hill, NC 27599 7352, USA
    Curr Gene Ther 10:319-40. 2010
    ..Continued work in these areas should synergize strategies to improve capsids and transgene cassettes that will eventually lead to optimized vectors ideally suited for translational success...
  2. ncbi request reprint Biosafety of Recombinant Adeno-associated Virus Vectors
    David J Dismuke
    UNC Gene Therapy Center, University of North Carolina at Chapel Hill, 7119 Thurston Bowles Building, 104 Manning Drive Campus Box 7352, Chapel Hill, NC 27599 7352, USA
    Curr Gene Ther 13:434-52. 2013
    ....
  3. pmc AAV exploits subcellular stress associated with inflammation, endoplasmic reticulum expansion, and misfolded proteins in models of cystic fibrosis
    Jarrod S Johnson
    Department of Pharmacology, University of North Carolina, Chapel Hill, North Carolina, United States of America
    PLoS Pathog 7:e1002053. 2011
    ....
  4. pmc Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles
    Wuping Li
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 16:1252-60. 2008
    ..Application of this technology to alternative cell/tissue types using AAV or other viral capsid sequences is likely to yield a new class of biological nanoparticles as vectors for human gene transfer...
  5. doi request reprint Efficient retrograde neuronal transduction utilizing self-complementary AAV1
    Edmund R Hollis
    Department of Neurosciences, University of California, San Diego, La Jolla, California 92093, USA
    Mol Ther 16:296-301. 2008
    ..91 +/- 0.24% of lumbar MNs were transduced. Our data provide the basis for increased retrograde transduction efficiency using peripheral injections of scAAV1 vectors for therapeutic gene delivery to the spinal cord...
  6. ncbi request reprint Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose
    Zhijian Wu
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 16:280-9. 2008
    ....
  7. ncbi request reprint Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications
    Joshua C Grieger
    University of North Carolina Gene Therapy Center, Chapel Hill 27599, USA
    Adv Biochem Eng Biotechnol 99:119-45. 2005
    ..In this chapter, we will provide a comprehensive overview of the recent advances in rAAV vector production and purification, vector development, and clinical applications...
  8. ncbi request reprint Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells
    TERESA BORRAS
    Department of Ophthalmology, University of North Carolina School of Medicine, 103 Mason Farm Road, Chapel Hill, NC 27599, USA
    J Gene Med 8:589-602. 2006
    ..Because of the importance of regulating elevated IOP by long-term gene therapy, we investigated mechanisms of AAV transduction to the human trabecular meshwork (TM)...
  9. ncbi request reprint Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency
    Ulrich T Hacker
    Klinik fur Innere Medizin I, Klinikum der Universität zu Köln, Joseph Stelzmann Strasse 9, 50925 Köln, Germany
    J Gene Med 7:1429-38. 2005
    ..Different adeno-associated virus (AAV) serotypes have been characterized so far, which show considerable differences in tissue tropism. Consequently, we aimed to characterize the most efficient serotype for this application...
  10. pmc Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors
    Sifeng Chen
    Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA
    Hum Gene Ther 16:235-47. 2005
    ..These results suggest the unique potential of rAAV1 and rAAV5-based vectors for vascular-targeted gene-based therapeutic strategies...
  11. ncbi request reprint Autoimmune anemia in macaques following erythropoietin gene therapy
    Pierre Chenuaud
    Institut National de la Sante et de la Recherche Medicale, Nantes, France
    Blood 103:3303-4. 2004
    ..It raises some concerns when a therapeutic protein is produced at high levels from an ectopic site...
  12. ncbi request reprint Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain
    Haiyan Fu
    Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, USA
    Mol Ther 8:911-7. 2003
    ....
  13. ncbi request reprint Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery
    Haiyan Fu
    Division of Genetics and Metabolism, Department of Pediatrics, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
    Mol Ther 5:42-9. 2002
    ..5 mm) in a 6-month duration of experiments. These results provide a basis for the development of a treatment for neurological disease in MPS IIIB patients using AAV vectors...
  14. pmc Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps
    Joshua C Grieger
    Curriculm in Genetics and Molecular Biology and Gene Therapy Center, University of North Carolina at Chapel Hill, 27599 7352, USA
    J Virol 79:9933-44. 2005
    ..0 kb but the larger genome-containing virions are preferentially degraded by the proteasome and that this block can be overcome by the addition of proteasome inhibitors...
  15. pmc Separate basic region motifs within the adeno-associated virus capsid proteins are essential for infectivity and assembly
    Joshua C Grieger
    Curriculum in Genetics and Molecular Biology, Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7352, USA
    J Virol 80:5199-210. 2006
    ..This study establishes the importance of two BR motifs on the AAV2 capsid that are essential for infectivity and virion assembly...
  16. pmc Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry
    Aravind Asokan
    Gene Therapy Center, 7113 Thurston Building, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7352, USA
    J Virol 80:8961-9. 2006
    ..The aforementioned results suggest that the internalization of AAV2 in 293 cells might follow a "click-to-fit" mechanism that involves the cooperative binding of heparan sulfate and alpha5beta1 integrin by the AAV2 capsids...
  17. ncbi request reprint From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop
    Arvind Asokan
    Gene Therapy Center, The University of North Carolina at Chapel Hill, NC 27599, USA
    Mol Ther 11:656-60. 2005

Research Grants21

  1. AAV Gene Therapy for AAT deficiency
    RICHARD SAMULSKI; Fiscal Year: 2009
    ..These new variants will be generated using an approach with in vitro "DNA shuffling" and in vivo selection in liver. This study would be very important to design AAV/AAT vector for future human clinical trials. ..
  2. Rational and combinatorial engineering of AAV vectors
    Richard J Samulski; Fiscal Year: 2010
    ....
  3. CTL response to AAV Vector
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
    ....
  4. Differential regulation of multiple transgenes for treatment of eye disease
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
    ....
  5. AAV Gene Therapy for AAT deficiency
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
    ..These new variants will be generated using an approach with in vitro "DNA shuffling" and in vivo selection in liver. This study would be very important to design AAV/AAT vector for future human clinical trials. ..
  6. CTL response to AAV Vector
    Chengwen Li; Fiscal Year: 2011
    ....
  7. Rational and combinatorial engineering of AAV vectors
    Richard J Samulski; Fiscal Year: 2011
    ....
  8. MUSCLE DELIVERY OF AAV VECTORS FOR GENE THERAPY
    RICHARD SAMULSKI; Fiscal Year: 2001
    ....
  9. rAAV Revitalized Structural Allografts
    RICHARD SAMULSKI; Fiscal Year: 2005
    ..Thus, our success here will open many areas of investigation and opportunities in all areas of medicine. ..
  10. Muscle-specific AAV Vectors for DMD Gene Therapy
    RICHARD SAMULSKI; Fiscal Year: 2007
    ..The novel reagents developed and optimized in this proposal will be advanced for further therapeutic testing in larger animal models of muscular dystrophy and eventually in human clinical trials in the near future. ..