Adeno-associated vectors for gene delivery to the nervous system

PJ Mulcahy, C Binny, B Muszynski, E Karyka… - Gene delivery and …, 2015 - Springer
Gene therapy approaches based on adeno-associated viral vector (AAV) systems offer
many unique advantages for nervous system applications. These vectors are opening up …

Production of Recombinant Adeno‐Associated Viral Vectors and Use in In Vitro and In Vivo Administration

RA Haberman, G Kroner‐Lux… - Current protocols in …, 1999 - Wiley Online Library
Adeno‐associated virus (AAV) vectors are able to transduce CNS cells in vitro and in vivo for
extended periods of time without immune complications. The ability to efficiently deliver …

Adeno-associated virus vectors for gene therapy of neurodegenerative disorders.

MJ During, P Leone - Clinical Neuroscience (New York, NY), 1995 - europepmc.org
Adeno-associated virus (AAV) shows significant potential as a gene delivery system.
Although it is ubiquitous in its distribution, with approximately 85% of the adult population in …

Adeno-associated viral vectors as agents for gene delivery: application in disorders and trauma of the central nervous system

MJ Ruitenberg, R Eggers, GJ Boer, J Verhaagen - Methods, 2002 - Elsevier
The use of viral vectors as agents for gene delivery provides a direct approach to manipulate
gene expression in the mammalian central nervous system (CNS). The present article …

Current development of adeno-associated viral vectors.

G Romano - Drug news & perspectives, 2005 - europepmc.org
Vectors based on adeno-associated virus (AAV) have recently been used in phase I clinical
trials for the treatment of neurological disorders, such as Parkinson's and Canavan's …

Adeno-associated virus vectors: activity and applications in the CNS

AL Peel, RL Klein - Journal of neuroscience methods, 2000 - Elsevier
Transgenic strategies are useful for funcional studies and they may also lead to novel
therapies. Controlling transgene expression in defined cell populations over time is …

Transduction of the central nervous system after intracerebroventricular injection of adeno-associated viral vectors in neonatal and juvenile mice

S Gholizadeh, S Tharmalingam… - Human gene therapy …, 2013 - liebertpub.com
Several neurodevelopmental and neurodegenerative disorders affecting the central nervous
system are potentially treatable via viral vector-mediated gene transfer. Adeno-associated …

Adeno-associated virus vectors for gene transfer to the brain

T Okada, T Nomoto, K Shimazaki, W Lijun, Y Lu… - Methods, 2002 - Elsevier
Gene therapy is a novel method under investigation for the treatment of neurological
disorders. Considerable interest has focused on the possibility of using viral vectors to …

Adeno-associated virus vectors and neurological gene therapy

DS Ojala, DP Amara, DV Schaffer - The Neuroscientist, 2015 - journals.sagepub.com
Gene therapy has strong potential for treating a variety of genetic disorders, as
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …

Adeno associated viral vectors for the nervous system

BK Kaspar, FH Gage - Gene therapy for neurological …, 2016 - api.taylorfrancis.com
The realization of gene therapy for the central nervous system has advanced to the realmsof
clinical practice. The brain and spinal cord, once inaccessible to certain therapies …