Literature DB >> 25238277

Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.

Principal Investigators Barry J Byrne1, Shelley Collins, Coinvestigators Cathryn Mah, Barbara Smith, Thomas Conlon, Subinvestigators Danny Martin, Manuela Corti, Brian Cleaver, Saleem Islam, Lee Ann Lawson.   

Abstract

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Year:  2014        PMID： 25238277     DOI： 10.1089/humc.2014.2514

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


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  21 in total

1.  Pompe disease: how to solve many problems with one solution.

Authors:  Rosa Puertollano; Nina Raben
Journal:  Ann Transl Med       Date:  2018-08

Review 2.  Pompe Disease: From Basic Science to Therapy.

Authors:  Lara Kohler; Rosa Puertollano; Nina Raben
Journal:  Neurotherapeutics       Date:  2018-10       Impact factor: 7.620

3.  Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Authors:  Francesco Puzzo; Pasqualina Colella; Maria G Biferi; Deeksha Bali; Nicole K Paulk; Patrice Vidal; Fanny Collaud; Marcelo Simon-Sola; Severine Charles; Romain Hardet; Christian Leborgne; Amine Meliani; Mathilde Cohen-Tannoudji; Stephanie Astord; Bernard Gjata; Pauline Sellier; Laetitia van Wittenberghe; Alban Vignaud; Florence Boisgerault; Martine Barkats; Pascal Laforet; Mark A Kay; Dwight D Koeberl; Giuseppe Ronzitti; Federico Mingozzi
Journal:  Sci Transl Med       Date:  2017-11-29       Impact factor: 17.956

Review 4.  Next Generation of Adeno-Associated Virus Vectors for Gene Therapy for Human Liver Diseases.

Authors:  Kenneth I Berns; Arun Srivastava
Journal:  Gastroenterol Clin North Am       Date:  2019-04-01       Impact factor: 3.806

Review 5.  In vivo tissue-tropism of adeno-associated viral vectors.

Authors:  Arun Srivastava
Journal:  Curr Opin Virol       Date:  2016-09-03       Impact factor: 7.090

Review 6.  Progress and challenges of gene therapy for Pompe disease.

Authors:  Giuseppe Ronzitti; Fanny Collaud; Pascal Laforet; Federico Mingozzi
Journal:  Ann Transl Med       Date:  2019-07

Review 7.  Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy.

Authors:  Barry J Byrne; David D Fuller; Barbara K Smith; Nathalie Clement; Kirsten Coleman; Brian Cleaver; Lauren Vaught; Darin J Falk; Angela McCall; Manuela Corti
Journal:  Ann Transl Med       Date:  2019-07

Review 8.  [Innovative therapeutic approaches for hereditary neuromuscular diseases].

Authors:  J Kirschner; B Schoser
Journal:  Nervenarzt       Date:  2018-10       Impact factor: 1.214

9.  Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.

Authors:  Manuela Corti; Cristina Liberati; Barbara K Smith; Lee Ann Lawson; Ibrahim S Tuna; Thomas J Conlon; Kirsten E Coleman; Saleem Islam; Roland W Herzog; David D Fuller; Shelley W Collins; Barry J Byrne
Journal:  Hum Gene Ther Clin Dev       Date:  2017-12       Impact factor: 5.032

10.  Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.

Authors:  Phillip A Doerfler; Adrian G Todd; Nathalie Clément; Darin J Falk; Sushrusha Nayak; Roland W Herzog; Barry J Byrne
Journal:  Hum Gene Ther       Date:  2016-01       Impact factor: 5.695
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