Genetic fate of recombinant adeno-associated virus vector genomes in muscle

BC Schnepp, KR Clark, DL Klemanski… - Journal of …, 2003 - Am Soc Microbiol
Recombinant adeno-associated virus (rAAV) vectors are promising human gene transfer
vectors, because they mediate long-term gene expression in vivo. The vector DNA form …

Specific depletion of human anti-adenovirus antibodies facilitates transduction in an in vivo model for systemic gene therapy

A Rahman, V Tsai, A Goudreau, JY Shinoda, SF Wen… - Molecular Therapy, 2001 - cell.com
Recombinant adenoviral (rAd) vectors are capable of mediating high-efficiency gene
transfer in vivo. Under conditions requiring systemic administration, however, the use of rAd …

Gene transfer into the CNS using recombinant adeno-associated virus: analysis of vector DNA forms resulting in sustained expression

KR Clark, TJ Sferra, W Lo, G Qu, R Chen… - Journal of drug …, 1999 - Taylor & Francis
Recombinant adeno-associated virus (rAAV) vectors have shown significant promise as
vehicles for in vivo gene transfer, particularly for transduction of organs composed primarily …

Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector–mediated gene therapy

VM Velazquez, DG Bowen… - Blood, The Journal of the …, 2009 - ashpublications.org
Recombinant adeno-associated virus (rAAV) vectors are considered promising for human
gene replacement because they facilitate stable expression of therapeutic proteins in …

Tetracycline-regulatable adenovirus vectors: pharmacologic properties and clinical potential

S Nakagawa, B Massie, RG Hawley - European journal of pharmaceutical …, 2001 - Elsevier
Stringent control of gene expression in human gene therapy strategies is important for both
therapeutic and safety reasons. Replication-defective vectors derived from adenoviruses …

Induction of T-cell infiltration and programmed death ligand 2 expression by adeno-associated virus in rhesus macaque skeletal muscle and modulation by …

ML Cramer, G Shao, LR Rodino-Klapac… - Human Gene …, 2017 - liebertpub.com
Use of adeno-associated virus (AAV) to transduce genes into skeletal muscles can be
associated with T-cell responses to viral capsid and/or to transgenic protein. Intramuscular …

A new method for recombinant adeno-associated virus vector delivery to murine diaphragm

C Mah, TJ Fraites, KO Cresawn, I Zolotukhin… - Molecular Therapy, 2004 - cell.com
Genetically modified mice are important models for evaluation of potential gene therapies for
human diseases. However, their small size often precludes the use of clinically feasible …

Long-lasting adenovirus transgene expression in mice through neonatal intrathymic tolerance induction without the use of immunosuppression

RP DeMatteo, G Chu, M Ahn, E Chang… - Journal of …, 1997 - Am Soc Microbiol
The major barrier to the clinical application of adenovirus gene therapy for diseases that
require stable transgene expression is the immunogenicity of recombinant adenovirus …

Ovine adenovirus vectors overcome preexisting humoral immunity against human adenoviruses in vivo

C Hofmann, P Löser, G Cichon, W Arnold… - Journal of …, 1999 - Am Soc Microbiol
Recombinant human adenoviruses (hAd) have become widely used as tools to achieve
efficient gene transfer. However, successful application of hAd-derived vectors in clinical …

Regulated expression of erythropoietin from an AAV vector safely improves the anemia of β-thalassemia in a mouse model

J Johnston, J Tazelaar, VM Rivera, T Clackson… - Molecular therapy, 2003 - cell.com
In vivo gene transfer is being considered in the systemic delivery of therapeutic proteins.
This report evaluates the use of AAV vectors administered into muscle to deliver …