Literature DB >> 16412695

Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.

Ronald J Mandel1, Fredric P Manfredsson, Kevin D Foust, Aaron Rising, Sharon Reimsnider, Kevin Nash, Corinna Burger.   

Abstract

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 long-term transgene expression in the nervous system. These properties make rAAV a reasonable candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five early stage clinical trials for various neurodegenerative disorders. Therefore, we will review the currently available preclinical data using rAAV in animal models of central nervous system (CNS) disorders. Moreover, potential caveats for rAAV-based gene therapy in the CNS are also presented.

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Year:  2006        PMID： 16412695     DOI： 10.1016/j.ymthe.2005.11.009

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


  49 in total

1.  Optimal promoter usage for lentiviral vector-mediated transduction of cultured central nervous system cells.

Authors:  Mingjie Li; Nada Husic; Ying Lin; Heather Christensen; Ibrahim Malik; Sally McIver; Christine M LaPash Daniels; David A Harris; Paul T Kotzbauer; Mark P Goldberg; B Joy Snider
Journal:  J Neurosci Methods       Date:  2010-03-27       Impact factor: 2.390

2.  Serotype-dependent transduction efficiencies of recombinant adeno-associated viral vectors in monkey neocortex.

Authors:  Annelies Gerits; Pascaline Vancraeyenest; Samme Vreysen; Marie-Eve Laramée; Annelies Michiels; Rik Gijsbers; Chris Van den Haute; Lieve Moons; Zeger Debyser; Veerle Baekelandt; Lutgarde Arckens; Wim Vanduffel
Journal:  Neurophotonics       Date:  2015-10-01       Impact factor: 3.593

3.  Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.

Authors:  Kai Qiu; Darin J Falk; Paul J Reier; Barry J Byrne; David D Fuller
Journal:  Mol Ther       Date:  2011-10-18       Impact factor: 11.454

4.  AAV2/9-mediated overexpression of MIF inhibits SOD1 misfolding, delays disease onset, and extends survival in mouse models of ALS.

Authors:  Marcel F Leyton-Jaimes; Joy Kahn; Adrian Israelson
Journal:  Proc Natl Acad Sci U S A       Date:  2019-07-01       Impact factor: 11.205

Review 5.  Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions.

Authors:  Pedro R Lowenstein; Ronald J Mandel; Wei-Dong Xiong; Kurt Kroeger; Maria G Castro
Journal:  Curr Gene Ther       Date:  2007-10       Impact factor: 4.391

Review 6.  Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production.

Authors:  Ronald J Mandel; Corinna Burger; Richard O Snyder
Journal:  Exp Neurol       Date:  2007-08-24       Impact factor: 5.330

7.  Activation of virus uptake through induction of macropinocytosis with a novel polymerizing peptide.

Authors:  Sarah I Daniels; Erin E Soule; Katharine S Davidoff; John G Bernbaum; Duosha Hu; Kenji Maeda; Stephen J Stahl; Nicole E Naiman; Abdul A Waheed; Eric O Freed; Paul Wingfield; Robert Yarchoan; David A Davis
Journal:  FASEB J       Date:  2013-10-04       Impact factor: 5.191

Review 8.  Large animal models of neurological disorders for gene therapy.

Authors:  Christine Gagliardi; Bruce A Bunnell
Journal:  ILAR J       Date:  2009

9.  Adeno-associated viral (AAV) serotype 5 vector mediated gene delivery of endothelin-converting enzyme reduces Abeta deposits in APP + PS1 transgenic mice.

Authors:  Niki C Carty; Kevin Nash; Daniel Lee; Mary Mercer; Paul E Gottschall; Craig Meyers; Nicholas Muzyczka; Marcia N Gordon; Dave Morgan
Journal:  Mol Ther       Date:  2008-07-29       Impact factor: 11.454

10.  Brain delivery of AAV9 expressing an anti-PrP monovalent antibody delays prion disease in mice.

Authors:  Fabio Moda; Chiara Vimercati; Ilaria Campagnani; Margherita Ruggerone; Giorgio Giaccone; Michela Morbin; Lorena Zentilin; Mauro Giacca; Ileana Zucca; Giuseppe Legname; Fabrizio Tagliavini
Journal:  Prion       Date:  2012-07-30       Impact factor: 3.931
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