Systemic delivery of adeno-associated viral vectors

Current Opinion in Virology

Volumn 21, Issue , 2016, Pages 16-25

  Duan, Dongsheng ^a  

^a UNIVERSITY OF MISSOURI   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR; DRUG CARRIER;

BIOENGINEERING; GENE TRANSFER; NONHUMAN; PRIORITY JOURNAL; REVIEW; SEROTYPE; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID; ADMINISTRATION AND DOSAGE; ANIMAL; CLINICAL TRIAL (TOPIC); DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; INTRAVENOUS DRUG ADMINISTRATION; PRECLINICAL STUDY; PROCEDURES;

ADMINISTRATION, INTRAVENOUS; ANIMALS; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DRUG CARRIERS; DRUG EVALUATION, PRECLINICAL; GENETIC THERAPY; GENETIC VECTORS; HUMANS;

EID: 84978883265     PISSN: 18796257     EISSN: 18796265     Source Type: Journal    
DOI: 10.1016/j.coviro.2016.07.006     Document Type: Review

References (146)

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82. 82• Childers, M.K., Joubert, R., Poulard, K., Moal, C., Grange, R.W., Doering, J.A., Lawlor, M.W., Rider, B.E., Jamet, T., Daniele, N., et al. Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med, 6, 2014, 220ra210 This study suggests that isolated limb perfusion can result in bodywide improvement if the therapeutic product is an enzyme. The authors also showed that intravenous delivery of AAV-8 did not induce any immune response in the canine model of myotubular myopathy.
83. 83•• Yue, Y., Pan, X., Hakim, C.H., Kodippili, K., Zhang, K., Shin, J.H., Yang, H.T., McDonald, T., Duan, D., Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus. Hum Mol Genet 24 (2015), 5880–5890 This is the first study demonstraing successful systemic AAV delivery in young adult subjects in a large animal model of human diseases. This study sets the foundation for conducting systemic AAV therapy in boys afflicted by Duchenne muscular dystrophy.
84. 84 Hakim, C.H., Pan, X., Kodippili, K., Blessa, T., Yang, H.T., Yao, G., Leach, S., Emter, C., Yue, Y., Zhang, K., et al. Intravenous delivery of a novel micro-dystrophin vector prevented muscle deterioration in young adult canine Duchenne muscular dystrophy dogs. Mol Ther 24 (2016), S198–S199.
85. 14 for 4 months to 2 years. The results of this ongoing study suggest that systemic AAV gene therapy is safe and may dramatically change the disease course. This study opens the door for systemic AAV gene therapy for other diseases in human patients.
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136. 136 Huang, L.Y., Halder, S., Agbandje-McKenna, M., Parvovirus glycan interactions. Curr Opin Virol 7 (2014), 108–118.
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