Literature DB >> 29596011

Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space.

Dan Wang1,2,3, Jia Li1,2, Karen Tran1,2, Daniel R Burt1,2, Li Zhong1,2,3, Guangping Gao1,2,3,4,5.   

Abstract

Recombinant adeno-associated viruses (rAAVs) are the leading in vivo gene delivery platform, and have been extensively studied in gene therapy targeting various tissues, including the central nervous system (CNS). A single-bolus rAAV injection to the cerebrospinal fluid (CSF) space has been widely used to target the CNS, but it suffers from several drawbacks, such as leakage to peripheral tissues. Here, a protocol is described using an osmotic pump to infuse rAAV slowly into the mouse CSF space. Compared to the single-bolus injection technique, pump infusion can lead to higher CNS transduction and lower transduction in the peripheral tissues.

Entities:  

Keywords:  adeno-associated virus; central nervous system; gene therapy; intrathecal delivery

Mesh：

Year:  2018        PMID： 29596011      PMCID： PMC5910051          DOI： 10.1089/hgtb.2017.250

Source DB:  PubMed          Journal:  Hum Gene Ther Methods        ISSN： 1946-6536            Impact factor:   2.396


  40 in total

1.  Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector.

Authors:  S S Elliger; C A Elliger; C P Aguilar; N R Raju; G L Watson
Journal:  Gene Ther       Date:  1999-06       Impact factor: 5.250

2.  Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Authors:  Christian Hinderer; Peter Bell; Brittney L Gurda; Qiang Wang; Jean-Pierre Louboutin; Yanqing Zhu; Jessica Bagel; Patricia O'Donnell; Tracey Sikora; Therese Ruane; Ping Wang; Mark E Haskins; James M Wilson
Journal:  Mol Ther       Date:  2014-07-16       Impact factor: 11.454

3.  AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction.

Authors:  Lluis Samaranch; Waldy San Sebastian; Adrian P Kells; Ernesto A Salegio; Gregory Heller; John R Bringas; Philip Pivirotto; Stephen DeArmond; John Forsayeth; Krystof S Bankiewicz
Journal:  Mol Ther       Date:  2013-11-21       Impact factor: 11.454

4.  Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.

Authors:  Kathrin Meyer; Laura Ferraiuolo; Leah Schmelzer; Lyndsey Braun; Vicki McGovern; Shibi Likhite; Olivia Michels; Alessandra Govoni; Julie Fitzgerald; Pablo Morales; Kevin D Foust; Jerry R Mendell; Arthur H M Burghes; Brian K Kaspar
Journal:  Mol Ther       Date:  2014-10-31       Impact factor: 11.454

5.  Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.

Authors:  Virginia Haurigot; Sara Marcó; Albert Ribera; Miguel Garcia; Albert Ruzo; Pilar Villacampa; Eduard Ayuso; Sònia Añor; Anna Andaluz; Mercedes Pineda; Gemma García-Fructuoso; Maria Molas; Luca Maggioni; Sergio Muñoz; Sandra Motas; Jesús Ruberte; Federico Mingozzi; Martí Pumarola; Fatima Bosch
Journal:  J Clin Invest       Date:  2013-07-01       Impact factor: 14.808

6.  A Single Injection of Recombinant Adeno-Associated Virus into the Lumbar Cistern Delivers Transgene Expression Throughout the Whole Spinal Cord.

Authors:  Yansu Guo; Dan Wang; Tao Qiao; Chunxing Yang; Qin Su; Guangping Gao; Zuoshang Xu
Journal:  Mol Neurobiol       Date:  2015-06-07       Impact factor: 5.590

7.  Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

Authors:  Hongyan Wang; Bin Yang; Linghua Qiu; Chunxing Yang; Joshua Kramer; Qin Su; Yansu Guo; Robert H Brown; Guangping Gao; Zuoshang Xu
Journal:  Hum Mol Genet       Date:  2013-09-18       Impact factor: 6.150

8.  Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.

Authors:  J Hordeaux; L Dubreil; C Robveille; J Deniaud; Q Pascal; B Dequéant; J Pailloux; L Lagalice; M Ledevin; C Babarit; P Costiou; F Jamme; M Fusellier; Y Mallem; C Ciron; C Huchet; C Caillaud; M-A Colle
Journal:  Acta Neuropathol Commun       Date:  2017-09-06       Impact factor: 7.801

9.  SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice.

Authors:  Elisabeth Dirren; Julianne Aebischer; Cylia Rochat; Christopher Towne; Bernard L Schneider; Patrick Aebischer
Journal:  Ann Clin Transl Neurol       Date:  2015-01-15       Impact factor: 4.511

10.  Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

Authors:  Brittney L Gurda; Adrien De Guilhem De Lataillade; Peter Bell; Yanqing Zhu; Hongwei Yu; Ping Wang; Jessica Bagel; Charles H Vite; Tracey Sikora; Christian Hinderer; Roberto Calcedo; Alexander D Yox; Richard A Steet; Therese Ruane; Patricia O'Donnell; Guangping Gao; James M Wilson; Margret Casal; Katherine P Ponder; Mark E Haskins
Journal:  Mol Ther       Date:  2015-10-08       Impact factor: 11.454
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  2 in total

1.  Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy.

Authors:  Yi Gong; Anna Berenson; Fiza Laheji; Guangping Gao; Dan Wang; Carrie Ng; Adrienn Volak; Rene Kok; Vasileios Kreouzis; Inge M Dijkstra; Stephan Kemp; Casey A Maguire; Florian Eichler
Journal:  Hum Gene Ther       Date:  2018-12-18       Impact factor: 5.695

2.  Megalencephalic Leukoencephalopathy: Insights Into Pathophysiology and Perspectives for Therapy.

Authors:  Assumpció Bosch; Raúl Estévez
Journal:  Front Cell Neurosci       Date:  2021-01-22       Impact factor: 5.505
  2 in total

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