Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders
RJ Mandel, FP Manfredsson, KD Foust, A Rising… - Molecular Therapy, 2006 - cell.com
Recombinant adeno-associated virus (rAAV) is derived from a small human parvovirus with
an excellent safety profile. In addition, this viral vector efficiently transduces and supports …
an excellent safety profile. In addition, this viral vector efficiently transduces and supports …
Gene transfer by adeno-associated virus vectors into the central nervous system
X Xiao, J Li, TJ McCown, RJ Samulski - Experimental neurology, 1997 - Elsevier
Adeno-associated virus (AAV) vectors are derived from a nonpathogenic and defective
human parvovirus. Although currently unable to display the integration specificity featured by …
human parvovirus. Although currently unable to display the integration specificity featured by …
Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain
C Wang, CM Wang, KR Clark, TJ Sferra - Gene therapy, 2003 - nature.com
Recombinant adeno-associated virus serotype 2 (rAAV2) vectors have shown promise as
therapeutic agents for neurologic disorders. However, intracerebral administration of this …
therapeutic agents for neurologic disorders. However, intracerebral administration of this …
Adeno‐associated virus‐mediated gene delivery approaches for the treatment of CNS disorders
D Terzi, V Zachariou - Biotechnology Journal: Healthcare …, 2008 - Wiley Online Library
Over the last few years, a large number of preclinical and clinical studies have demonstrated
the potential of gene therapy applications using adeno‐associated viral (AAV) vectors. Gene …
the potential of gene therapy applications using adeno‐associated viral (AAV) vectors. Gene …
Adeno-associated virus as a delivery vector for gene therapy of human diseases
JH Wang, DJ Gessler, W Zhan, TL Gallagher… - Signal Transduction and …, 2024 - nature.com
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …
Recombinant adeno-associated viral vectors in the nervous system
Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene
delivery vector for the nervous system. It targets primarily neurons in the nervous system and …
delivery vector for the nervous system. It targets primarily neurons in the nervous system and …
Gene therapy for neurological disorders: progress and prospects
Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …
Immunological aspects of recombinant adeno-associated virus delivery to the mammalian brain
MY Mastakov, K Baer, CW Symes… - Journal of …, 2002 - Am Soc Microbiol
Recombinant adeno-associated viruses (rAAV) are highly efficient vectors for gene delivery
into the central nervous system (CNS). However, host inflammatory and immune responses …
into the central nervous system (CNS). However, host inflammatory and immune responses …
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 …
disorders. Considerable interest has focused on the possibility of using viral vectors to …
AAV production everywhere: a simple, fast, and reliable protocol for in‐house AAV vector production based on chloroform extraction
Recombinant adeno‐associated virus (rAAV) is a mammalian virus that has been altered to
be used as a gene delivery vehicle. Several changes to the viral genome have made them …
be used as a gene delivery vehicle. Several changes to the viral genome have made them …