Literature DB >> 29503193

Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products.

Barry J Byrne1.   

Abstract

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Mesh：

Year:  2018        PMID： 29503193      PMCID： PMC5911637          DOI： 10.1016/j.ymthe.2018.02.009

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


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  8 in total

1.  The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.

Authors:  Juliette Hordeaux; Qiang Wang; Nathan Katz; Elizabeth L Buza; Peter Bell; James M Wilson
Journal:  Mol Ther       Date:  2018-02-02       Impact factor: 11.454

2.  Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning.

Authors:  Manuela Corti; Brian Cleaver; Nathalie Clément; Thomas J Conlon; Kaitlyn J Faris; Gensheng Wang; Janet Benson; Alice F Tarantal; Davis Fuller; Roland W Herzog; Barry J Byrne
Journal:  Hum Gene Ther Clin Dev       Date:  2015-09       Impact factor: 5.032

3.  Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First.

Authors:  Terence R Flotte; Hildegard Büning
Journal:  Hum Gene Ther       Date:  2018-02-07       Impact factor: 5.695

4.  Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors:  Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal:  N Engl J Med       Date:  2017-11-02       Impact factor: 91.245

5.  Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.

Authors:  Christian Hinderer; Nathan Katz; Elizabeth L Buza; Cecilia Dyer; Tamara Goode; Peter Bell; Laura K Richman; James M Wilson
Journal:  Hum Gene Ther       Date:  2018-02-12       Impact factor: 5.695

6.  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

7.  Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain.

Authors:  Benjamin E Deverman; Piers L Pravdo; Bryan P Simpson; Sripriya Ravindra Kumar; Ken Y Chan; Abhik Banerjee; Wei-Li Wu; Bin Yang; Nina Huber; Sergiu P Pasca; Viviana Gradinaru
Journal:  Nat Biotechnol       Date:  2016-02-01       Impact factor: 54.908

Review 8.  Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies.

Authors:  Beatriz Silva Lima; Mafalda Ascensão Videira
Journal:  Mol Ther Methods Clin Dev       Date:  2018-01-31       Impact factor: 6.698
  8 in total
  4 in total

Review 1.  Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain.

Authors:  Charlotte J Sumner; Thomas O Crawford
Journal:  J Clin Invest       Date:  2018-07-09       Impact factor: 14.808

Review 2.  Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy.

Authors:  Nicola Specchio; Alessandro Ferretti; Marina Trivisano; Nicola Pietrafusa; Chiara Pepi; Costanza Calabrese; Susanna Livadiotti; Alessandra Simonetti; Paolo Rossi; Paolo Curatolo; Federico Vigevano
Journal:  Drugs       Date:  2021-01       Impact factor: 9.546

3.  Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.

Authors:  Jonathan B Rosenberg; Alvin Chen; Stephen M Kaminsky; Ronald G Crystal; Dolan Sondhi
Journal:  Expert Opin Orphan Drugs       Date:  2019-11-27       Impact factor: 0.694

Review 4.  Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.

Authors:  Albert M Maguire; Jean Bennett; Elena M Aleman; Bart P Leroy; Tomas S Aleman
Journal:  Mol Ther       Date:  2020-12-03       Impact factor: 11.454
  4 in total

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