Literature DB >> 26357010

Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.

Alisha M Gruntman1, Terence R Flotte1,2,3.   

Abstract

Recombinant adeno-associated virus (rAAV) can be delivered to the skeletal muscle of the limb (pelvic or thoracic) by means of regional intravascular delivery. This review summarizes the evolution of this technique to deliver rAAV either via the arterial blood supply or via the peripheral venous circulation. The focus of this review is on applications in large animal models, including preclinical studies. Based on this overview of past research, we aim to inform the design of preclinical and clinical studies.

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Year:  2015        PMID： 26357010      PMCID： PMC4606036          DOI： 10.1089/humc.2015.116

Source DB:  PubMed          Journal:  Hum Gene Ther Clin Dev        ISSN： 2324-8637            Impact factor:   5.032


  40 in total

1.  Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector.

Authors:  J P Greelish; L T Su; E B Lankford; J M Burkman; H Chen; S K Konig; I M Mercier; P R Desjardins; M A Mitchell; X G Zheng; J Leferovich; G P Gao; R J Balice-Gordon; J M Wilson; H H Stedman
Journal:  Nat Med       Date:  1999-04       Impact factor: 53.440

2.  Intraveneus regional analgesia. A useful method of producing analgesia of thelimbs.

Authors:  C M HOLMES
Journal:  Lancet       Date:  1963-02-02       Impact factor: 79.321

3.  The efficient expression of intravascularly delivered DNA in rat muscle.

Authors:  V Budker; G Zhang; I Danko; P Williams; J Wolff
Journal:  Gene Ther       Date:  1998-02       Impact factor: 5.250

4.  Optimization of regional intraarterial naked DNA-mediated transgene delivery to skeletal muscles in a large animal model.

Authors:  Gawiyou Danialou; Alain S Comtois; Stefan Matecki; Josephine Nalbantoglu; George Karpati; Renald Gilbert; Pascale Geoffroy; Sandra Gilligan; Jean-Francois Tanguay; Basil J Petrof
Journal:  Mol Ther       Date:  2005-02       Impact factor: 11.454

5.  Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer.

Authors:  Victor M Rivera; Guang-ping Gao; Rebecca L Grant; Michael A Schnell; Philip W Zoltick; Leonard W Rozamus; Tim Clackson; James M Wilson
Journal:  Blood       Date:  2004-10-26       Impact factor: 22.113

6.  Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route.

Authors:  W K Cho; S Ebihara; J Nalbantoglu; R Gilbert; B Massie; P Holland; G Karpati; B J Petrof
Journal:  Hum Gene Ther       Date:  2000-03-20       Impact factor: 5.695

7.  Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors.

Authors:  S Song; M Morgan; T Ellis; A Poirier; K Chesnut; J Wang; M Brantly; N Muzyczka; B J Byrne; M Atkinson; T R Flotte
Journal:  Proc Natl Acad Sci U S A       Date:  1998-11-24       Impact factor: 11.205

8.  Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.

Authors:  Valder R Arruda; Hansell H Stedman; Timothy C Nichols; Mark E Haskins; Matthew Nicholson; Roland W Herzog; Linda B Couto; Katherine A High
Journal:  Blood       Date:  2004-10-12       Impact factor: 22.113

9.  Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Authors:  Caroline Le Guiner; Marie Montus; Laurent Servais; Yan Cherel; Virginie Francois; Jean-Laurent Thibaud; Claire Wary; Béatrice Matot; Thibaut Larcher; Lydie Guigand; Maeva Dutilleul; Claire Domenger; Marine Allais; Maud Beuvin; Amélie Moraux; Johanne Le Duff; Marie Devaux; Nicolas Jaulin; Mickaël Guilbaud; Virginie Latournerie; Philippe Veron; Sylvie Boutin; Christian Leborgne; Diana Desgue; Jack-Yves Deschamps; Sophie Moullec; Yves Fromes; Adeline Vulin; Richard H Smith; Nicolas Laroudie; Frédéric Barnay-Toutain; Christel Rivière; Stéphanie Bucher; Thanh-Hoa Le; Nicolas Delaunay; Mehdi Gasmi; Robert M Kotin; Gisèle Bonne; Oumeya Adjali; Carole Masurier; Jean-Yves Hogrel; Pierre Carlier; Philippe Moullier; Thomas Voit
Journal:  Mol Ther       Date:  2014-08-04       Impact factor: 11.454

10.  High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.

Authors:  Zheng Fan; Keith Kocis; Robert Valley; James F Howard; Manisha Chopra; Yasheng Chen; Hongyu An; Weili Lin; Joseph Muenzer; William Powers
Journal:  Hum Gene Ther       Date:  2015-07-30       Impact factor: 5.695
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  2 in total

1.  The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute.

Authors:  Terence R Flotte; Eric Daniels; Janet Benson; Jeneé M Bevett-Rose; Kenneth Cornetta; Margaret Diggins; Julie Johnston; Susan Sepelak; Johannes C M van der Loo; James M Wilson; Cheryl L McDonald
Journal:  Hum Gene Ther Clin Dev       Date:  2017-11-27       Impact factor: 5.032

Review 2.  Calcium Mechanisms in Limb-Girdle Muscular Dystrophy with CAPN3 Mutations.

Authors:  Jaione Lasa-Elgarresta; Laura Mosqueira-Martín; Neia Naldaiz-Gastesi; Amets Sáenz; Adolfo López de Munain; Ainara Vallejo-Illarramendi
Journal:  Int J Mol Sci       Date:  2019-09-13       Impact factor: 5.923
  2 in total

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