AAVHSC 17 / Homology Medicines 
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  • ||||||||||  AAVHSC 17 / Homology Medicines
    Genome Editing by Clade F AAV Homology Donors in the Presence and Absence of Site-Specific DNA Breaks (ASGCT 2019) -  Apr 19, 2019 - Abstract #966; Author: Geoffrey L. Rogers, Hsu-Yu Chen, Heidy Morales, Paula M. Cannon Molecular Microbiology and Immunology, University of Southern California, Los Angeles, CA; Presentation Time: May 02, 2019; 10:30-10:45   
    (2018), whose expression would require insertion downstream of a promoter and splice donor (AAVS1-SA-2A-GFP). In K562 cells transduced with the CCR5-PGK-GFP series of vectors, all populations initially contained GFP+ cells, but rapidly lost GFP expression to be
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    Biodistribution of AAVHSCs in the central nervous system of non-human primates (Neuroscience 2018) -  Sep 20, 2018 - Abstract #610.27 / LLL24; Author: *J. GINGRAS1; K. OLIVIERI2; N. ZAPATA2; L. SMITH2; H. RUBIN2; P. MORALES3; J. ELLSWORTH2; A. SEYMOUR2; Presentation Time: November 06, 2018; 15:00-16:00   
    Both neuronal cell bodies, dendrites and axons /axonal tracts were detected. These data demonstrate that, like AAV9, AAVHSCs effectively cross the BBB following intravenous delivery in non-human primates, creating the potential for therapeutic applications in treating human genetic diseases of the CNS.
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    Transduction of Photoreceptor and Pigmented Epithelial Cells Following a Single Subretinal Injection of AAVHSC17 in Minipigs (ASGCT 2018) -  Jun 11, 2018 - Abstract #303; Author: Jeff L. Ellsworth; Hillard Rubin; Albert Seymour; Presentation Time: May 16, 2018; 17:30-19:30   
    Taken together, these data demonstrate that administration of AAVHSC17-CBA-scGFP by subretinal injection in minipigs was well-tolerated and resulted in GFP expression in all retinal layers. These studies suggest that AAVHSCs may be useful as therapeutic vectors for treating diseases of the eye in humans.
  • ||||||||||  AAVHSC 17 / Homology Medicines
    Journal:  Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans. (Pubmed Central) -  Apr 7, 2018   
    Significant cross-reactivity of NAbs across all three AAV serotypes was observed. These data show that approximately 80% of humans evaluated were seronegative for pre-existing NAbs to the AAV serotypes tested, suggesting that the vast majority of human subjects would be amenable to therapeutic intervention with Clade F AAVs.