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  1. No Access

    Article

    Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

    A recombinant adeno-associated virus (rAAV) vector carrying the human factor IX cDNA was tested for safety and therapeutic gene expression in a canine model of human hemophilia B. Intramuscular delivery of rAA...

    PE Monahan, RJ Samulski, J Tazelaar, X **ao, TC Nichols, DA Bellinger… in Gene Therapy (1998)

  2. No Access

    Article

    In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector

    An adeno-associated virus (AAV) vector, expressing genes for human tyrosine hydroxylase (TH) and aromatic amino acid decarboxylase (AADC), demonstrated significantly increased production of dopamine in 293 (hu...

    MJ During, RJ Samulski, JD Elsworth, MG Kaplitt, P Leone, X **ao, J Li… in Gene Therapy (1998)

  3. No Access

    Article

    Inducible long-term gene expression in brain with adeno-associated virus gene transfer

    Recombinant adeno-associated virus (rAAV) vectors hold promise for treating a number of neurological disorders due to the ability to deliver long-term gene expression without toxicity or immune response. Criti...

    RP Haberman, TJ McCown, RJ Samulski in Gene Therapy (1998)

  4. No Access

    Article

    Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

    Adeno-associated virus (AAV) vectors package single-stranded genomes and require host-cell synthesis of the complementary strand for transduction. However, when the genome is half wild-type size, AAV can packa...

    DM McCarty, PE Monahan, RJ Samulski in Gene Therapy (2001)