Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy.

Brain : a Journal of Neurology
Sea Young YoonJ H Wolfe

Abstract

Intravascular injection of certain adeno-associated virus vector serotypes can cross the blood-brain barrier to deliver a gene into the CNS. However, gene distribution has been much more limited within the brains of large animals compared to rodents, rendering this approach suboptimal for treatment of the global brain lesions present in most human neurogenetic diseases. The most commonly used serotype in animal and human studies is 9, which also has the property of being transported via axonal pathways to distal neurons. A small number of other serotypes share this property, three of which were tested intravenously in mice compared to 9. Serotype hu.11 transduced fewer cells in the brain than 9, rh8 was similar to 9, but hu.32 mediated substantially greater transduction than the others throughout the mouse brain. To evaluate the potential for therapeutic application of the hu.32 serotype in a gyrencephalic brain of larger mammals, a hu.32 vector expressing the green fluorescent protein reporter gene was evaluated in the cat. Transduction was widely distributed in the cat brain, including in the cerebral cortex, an important target since mental retardation is an important component of many of the human neurogenetic diseases. The...Continue Reading

References

Sep 29, 1999·Journal of Medical Virology·K ErlesJ R Schlehofer
May 28, 2004·Journal of Virology·Guangping GaoJames M Wilson
Feb 26, 2005·Annals of Neurology·Charles H ViteJohn H Wolfe
Jan 18, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Cassia N Cearley, John H Wolfe
Aug 7, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Douglas M McCarty
Aug 21, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Cassia N CearleyJohn H Wolfe
Dec 23, 2008·Nature Biotechnology·Kevin D FoustBrian K Kaspar
Jan 13, 2009·The Journal of Infectious Diseases·Roberto CalcedoJames M Wilson
Apr 16, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Sandra DuqueMartine Barkats
Jun 12, 2010·Science Translational Medicine·Chiara F ValoriMimoun Azzouz
Dec 2, 2010·Human Molecular Genetics·Elisa DominguezMartine Barkats
Mar 10, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Haiyan FuDouglas M McCarty
Apr 14, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Steven J GrayR Jude Samulski
Jul 22, 2011·Clinical and Vaccine Immunology : CVI·Roberto CalcedoJames M Wilson
Aug 4, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Adam K BevanBrian K Kaspar
Oct 29, 2011·Methods in Molecular Biology·Mavis Agbandje-McKenna, Jürgen Kleinschmidt
Dec 29, 2011·Human Gene Therapy·Lluis SamaranchKrystof S Bankiewicz
Apr 20, 2012·Journal of Virology·Christie L BellJames M Wilson
Apr 24, 2013·Nature Reviews. Neurology·Michele SimonatoJoseph C Glorioso
Jul 3, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Seemin Seher AhmedGuangping Gao
Jan 15, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·Lluis SamaranchKrystof S Bankiewicz
May 2, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·Bin YangGuangping Gao
Sep 12, 2015·Molecular Therapy : the Journal of the American Society of Gene Therapy·Sea Young YoonJohn H Wolfe
Oct 9, 2015·Molecular Therapy : the Journal of the American Society of Gene Therapy·Brittney L GurdaMark E Haskins
Nov 28, 2015·Methods in Molecular Biology·Michael J CastleJohn H Wolfe
Dec 17, 2015·Journal of Neuropathology and Experimental Neurology·Manoj KumarHarish Poptani
Dec 29, 2015·Molecular Therapy : the Journal of the American Society of Gene Therapy·Sourav R ChoudhuryMiguel Sena-Esteves
Feb 2, 2016·Nature Biotechnology·Benjamin E DevermanViviana Gradinaru
Nov 2, 2017·The New England Journal of Medicine·Jerry R MendellBrian K Kaspar
Feb 13, 2018·Molecular Therapy : the Journal of the American Society of Gene Therapy·Juliette HordeauxJames M Wilson
Aug 16, 2018·Molecular Therapy. Methods & Clinical Development·Eloise HudryLuk H Vandenberghe

❮ Previous
Next ❯

Citations

Jul 17, 2020·Brain : a Journal of Neurology·Ahad A Rahim, Paul Gissen
Sep 19, 2020·Brain : a Journal of Neurology·Dimitri M Kullmann
Dec 16, 2020·Molecular Genetics and Metabolism·Jitka MajovskaPetr Dusek
Jul 10, 2021·Frontiers in Cellular Neuroscience·Georg von JonquieresGary D Housley
Aug 20, 2021·Life Science Alliance·Cristin D DavidsonWilliam J Pavan

❮ Previous
Next ❯

Related Concepts

Related Feeds

Blood Brain Barrier Chips

The blood brain barrier (BBB) is comprised of endothelial cells that regulate the influx and outflux of plasma concentrations. Lab-on-a-chip devices allow scientists to model diseases and mechanisms such as the passage of therapeutic antibodies across the BBB. Discover the latest research on BBB chips here.

Barrel cortex

Here is the latest research on barrel cortex, a region of somatosensory and motor corticies in the brain, which are used by animals that rely on whiskers for world exploration.

Blood Brain Barrier

The blood brain barrier is a border that separates blood from cerebrospinal fluid. Discover the latest search on this highly selective semipermeable membrane here.

Related Papers

Molecular Therapy : the Journal of the American Society of Gene Therapy
Cassia N CearleyJ H Wolfe
International Journal of Molecular Sciences
Maria Rachele CeccariniT Beccari
Anales de pediatría : publicación oficial de la Asociación Española de Pediatría (A.E.P.)
F M Pérez FernándezA Chabas Bergón
© 2021 Meta ULC. All rights reserved