Literature DB >> 25136913

Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.

William W Hauswirth1.   

Abstract

Mesh：

Year:  2014        PMID： 25136913      PMCID： PMC4137346          DOI： 10.1089/hum.2014.2530

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


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  121 in total

1.  Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

Authors:  Frank M Dyka; Sanford L Boye; Vince A Chiodo; William W Hauswirth; Shannon E Boye
Journal:  Hum Gene Ther Methods       Date:  2014-04       Impact factor: 2.396

2.  Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.

Authors:  Rajeshwari D Koilkonda; Hong Yu; Tsung-Han Chou; William J Feuer; Marco Ruggeri; Vittorio Porciatti; David Tse; William W Hauswirth; Vince Chiodo; Sanford L Boye; Alfred S Lewin; Martha Neuringer; Lauren Renner; John Guy
Journal:  JAMA Ophthalmol       Date:  2014-04-01       Impact factor: 7.389

3.  Gene therapy rescues cone function in congenital achromatopsia.

Authors:  András M Komáromy; John J Alexander; Jessica S Rowlan; Monique M Garcia; Vince A Chiodo; Asli Kaya; Jacqueline C Tanaka; Gregory M Acland; William W Hauswirth; Gustavo D Aguirre
Journal:  Hum Mol Genet       Date:  2010-04-08       Impact factor: 6.150

4.  RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.

Authors:  Laurie L Molday; Hidayat Djajadi; Paul Yan; Lukasz Szczygiel; Sanford L Boye; Vince A Chiodo; Kevin Gregory-Evans; Marinko V Sarunic; William W Hauswirth; Robert S Molday
Journal:  Hum Mol Genet       Date:  2013-06-04       Impact factor: 6.150

5.  AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.

Authors:  Xufeng Dai; Juanjuan Han; Yan Qi; Hua Zhang; Lue Xiang; Jineng Lv; Jie Li; Wen-Tao Deng; Bo Chang; William W Hauswirth; Ji-jing Pang
Journal:  Invest Ophthalmol Vis Sci       Date:  2014-03-20       Impact factor: 4.799

6.  Nucleotide sequence evidence for rapid genotypic shifts in the bovine mitochondrial DNA D-loop.

Authors:  P D Olivo; M J Van de Walle; P J Laipis; W W Hauswirth
Journal:  Nature       Date:  1983 Nov 24-30       Impact factor: 49.962

7.  Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.

Authors:  V S Lopes; S E Boye; C M Louie; S Boye; F Dyka; V Chiodo; H Fofo; W W Hauswirth; D S Williams
Journal:  Gene Ther       Date:  2013-01-24       Impact factor: 5.250

8.  Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects.

Authors:  Karina E Guziewicz; Barbara Zangerl; András M Komáromy; Simone Iwabe; Vincent A Chiodo; Sanford L Boye; William W Hauswirth; William A Beltran; Gustavo D Aguirre
Journal:  PLoS One       Date:  2013-10-15       Impact factor: 3.240

9.  Next-generation sequencing of mitochondrial targeted AAV transfer of human ND4 in mice.

Authors:  Hong Yu; Arpit Mehta; Gaofeng Wang; William W Hauswirth; Vince Chiodo; Sanford L Boye; John Guy
Journal:  Mol Vis       Date:  2013-07-14       Impact factor: 2.367

10.  Effective delivery of large genes to the retina by dual AAV vectors.

Authors:  Ivana Trapani; Pasqualina Colella; Andrea Sommella; Carolina Iodice; Giulia Cesi; Sonia de Simone; Elena Marrocco; Settimio Rossi; Massimo Giunti; Arpad Palfi; Gwyneth J Farrar; Roman Polishchuk; Alberto Auricchio
Journal:  EMBO Mol Med       Date:  2013-12-15       Impact factor: 12.137
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  3 in total

1.  Effects of Altering HSPG Binding and Capsid Hydrophilicity on Retinal Transduction by AAV.

Authors:  Sean M Crosson; Antonette Bennett; Diego Fajardo; James J Peterson; Hangning Zhang; Wei Li; Matthew T Leahy; Colin K Jennings; Ryan F Boyd; Sanford L Boye; Mavis Agbandge-McKenna; Shannon E Boye
Journal:  J Virol       Date:  2021-03-03       Impact factor: 5.103

2.  Heparan Sulfate Binding Promotes Accumulation of Intravitreally Delivered Adeno-associated Viral Vectors at the Retina for Enhanced Transduction but Weakly Influences Tropism.

Authors:  Kenton T Woodard; Katharine J Liang; William C Bennett; R Jude Samulski
Journal:  J Virol       Date:  2016-10-14       Impact factor: 6.549

Review 3.  Gene-Based Therapeutics for Inherited Retinal Diseases.

Authors:  Beau J Fenner; Tien-En Tan; Amutha Veluchamy Barathi; Sai Bo Bo Tun; Sia Wey Yeo; Andrew S H Tsai; Shu Yen Lee; Chui Ming Gemmy Cheung; Choi Mun Chan; Jodhbir S Mehta; Kelvin Y C Teo
Journal:  Front Genet       Date:  2022-01-07       Impact factor: 4.599
  3 in total

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