Literature DB >> 29378415

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

Terence R Flotte1, Hildegard Büning2.   

Abstract

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Year:  2018        PMID： 29378415     DOI： 10.1089/hum.2018.021

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


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  12 in total

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

Authors:  Barry J Byrne
Journal:  Mol Ther       Date:  2018-03-01       Impact factor: 11.454

2.  AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset.

Authors:  David Goertsen; Nicholas C Flytzanis; Nick Goeden; Miguel R Chuapoco; Alexander Cummins; Yijing Chen; Yingying Fan; Qiangge Zhang; Jitendra Sharma; Yangyang Duan; Liping Wang; Guoping Feng; Yu Chen; Nancy Y Ip; James Pickel; Viviana Gradinaru
Journal:  Nat Neurosci       Date:  2021-12-09       Impact factor: 24.884

Review 3.  Advances in gene therapy for neurogenetic diseases: a brief review.

Authors:  Ying-Xuan Xie; Wen-Qi Lv; Yi-Kun Chen; Shunyan Hong; Xiang-Ping Yao; Wan-Jin Chen; Miao Zhao
Journal:  J Mol Med (Berl)       Date:  2021-11-27       Impact factor: 4.599

Review 4.  CRISPR technologies for the treatment of Duchenne muscular dystrophy.

Authors:  Eunyoung Choi; Taeyoung Koo
Journal:  Mol Ther       Date:  2021-04-03       Impact factor: 11.454

Review 5.  How will the field of gene therapy survive its success?

Authors:  William F Kaemmerer
Journal:  Bioeng Transl Med       Date:  2018-05-24

Review 6.  Lessons learned from lung and liver in-vivo gene therapy: implications for the future.

Authors:  Joost van Haasteren; Stephen C Hyde; Deborah R Gill
Journal:  Expert Opin Biol Ther       Date:  2018-08-10       Impact factor: 4.388

7.  New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina.

Authors:  Elizabeth M Simpson; Andrea J Korecki; Oriol Fornes; Trevor J McGill; Jorge Luis Cueva-Vargas; Jessica Agostinone; Rachelle A Farkas; Jack W Hickmott; Siu Ling Lam; Anthony Mathelier; Lauren M Renner; Jonathan Stoddard; Michelle Zhou; Adriana Di Polo; Martha Neuringer; Wyeth W Wasserman
Journal:  Hum Gene Ther       Date:  2018-10-02       Impact factor: 5.695

8.  The adeno-associated virus rh10 vector is an effective gene transfer system for chronic spinal cord injury.

Authors:  Yutaka Hoshino; Kenji Nishide; Narihito Nagoshi; Shinsuke Shibata; Nobuko Moritoki; Kota Kojima; Osahiko Tsuji; Morio Matsumoto; Jun Kohyama; Masaya Nakamura; Hideyuki Okano
Journal:  Sci Rep       Date:  2019-07-08       Impact factor: 4.379

9.  Systemic AAV6-synapsin-GFP administration results in lower liver biodistribution, compared to AAV1&2 and AAV9, with neuronal expression following ultrasound-mediated brain delivery.

Authors:  Danielle Weber-Adrian; Rikke Hahn Kofoed; Joseph Silburt; Zeinab Noroozian; Kairavi Shah; Alison Burgess; Shawna Rideout; Sebastian Kügler; Kullervo Hynynen; Isabelle Aubert
Journal:  Sci Rep       Date:  2021-01-21       Impact factor: 4.379

10.  AAV ablates neurogenesis in the adult murine hippocampus.

Authors:  Stephen Johnston; Sarah L Parylak; Stacy Kim; Nolan Mac; Christina Lim; Iryna Gallina; Cooper Bloyd; Alexander Newberry; Christian D Saavedra; Ondrej Novak; J Tiago Gonçalves; Fred H Gage; Matthew Shtrahman
Journal:  Elife       Date:  2021-07-14       Impact factor: 8.140
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