Literature DB >> 34327311

Stable Retinoid Analogue Targeted Dual pH-Sensitive Smart Lipid ECO/pDNA Nanoparticles for Specific Gene Delivery in the Retinal Pigment Epithelium.

Da Sun1, Rebecca M Schur1, Avery E Sears2, Song-Qi Gao1, Wenyu Sun1, Amirreza Naderi1, Timothy Kern3, Krzysztof Palczewski3, Zheng-Rong Lu1.   

Abstract

Entities:  

Year:  2020        PMID： 34327311      PMCID： PMC8317636          DOI： 10.1021/acsabm.0c00130

Source DB:  PubMed          Journal:  ACS Appl Bio Mater        ISSN： 2576-6422


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  40 in total

Review 1.  Adeno-associated virus-vectored gene therapy for retinal disease.

Authors:  Astra Dinculescu; Lyudmyla Glushakova; Seok-Hong Min; William W Hauswirth
Journal:  Hum Gene Ther       Date:  2005-06       Impact factor: 5.695

Review 2.  Chemistry of the retinoid (visual) cycle.

Authors:  Philip D Kiser; Marcin Golczak; Krzysztof Palczewski
Journal:  Chem Rev       Date:  2013-07-11       Impact factor: 60.622

3.  Molecular pharmacodynamics of emixustat in protection against retinal degeneration.

Authors:  Jianye Zhang; Philip D Kiser; Mohsen Badiee; Grazyna Palczewska; Zhiqian Dong; Marcin Golczak; Gregory P Tochtrop; Krzysztof Palczewski
Journal:  J Clin Invest       Date:  2015-06-15       Impact factor: 14.808

Review 4.  Emerging therapies for inherited retinal degeneration.

Authors:  Hendrik P N Scholl; Rupert W Strauss; Mandeep S Singh; Deniz Dalkara; Botond Roska; Serge Picaud; José-Alain Sahel
Journal:  Sci Transl Med       Date:  2016-12-07       Impact factor: 17.956

Review 5.  Gene therapy progress and prospects: the eye.

Authors:  J W B Bainbridge; M H Tan; R R Ali
Journal:  Gene Ther       Date:  2006-07-13       Impact factor: 5.250

Review 6.  Polyethylene glycol-conjugated copolymers for plasmid DNA delivery.

Authors:  Minhyung Lee; Sung Wan Kim
Journal:  Pharm Res       Date:  2005-01       Impact factor: 4.200

7.  Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi.

Authors:  Nadia R Ayat; Zhanhu Sun; Da Sun; Michelle Yin; Ryan C Hall; Amita M Vaidya; Xujie Liu; Andrew L Schilb; Josef H Scheidt; Zheng-Rong Lu
Journal:  Nucleic Acid Ther       Date:  2019-05-28       Impact factor: 5.486

8.  Mutations in the ABCA4 (ABCR) gene are the major cause of autosomal recessive cone-rod dystrophy.

Authors:  A Maugeri; B J Klevering; K Rohrschneider; A Blankenagel; H G Brunner; A F Deutman; C B Hoyng; F P Cremers
Journal:  Am J Hum Genet       Date:  2000-08-24       Impact factor: 11.025

9.  ABCA4 is an N-retinylidene-phosphatidylethanolamine and phosphatidylethanolamine importer.

Authors:  Faraz Quazi; Stepan Lenevich; Robert S Molday
Journal:  Nat Commun       Date:  2012-06-26       Impact factor: 14.919

10.  Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.

Authors:  Francesca Simonelli; Albert M Maguire; Francesco Testa; Eric A Pierce; Federico Mingozzi; Jeannette L Bennicelli; Settimio Rossi; Kathleen Marshall; Sandro Banfi; Enrico M Surace; Junwei Sun; T Michael Redmond; Xiaosong Zhu; Kenneth S Shindler; Gui-Shuang Ying; Carmela Ziviello; Carmela Acerra; J Fraser Wright; Jennifer Wellman McDonnell; Katherine A High; Jean Bennett; Alberto Auricchio
Journal:  Mol Ther       Date:  2009-12-01       Impact factor: 11.454
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  3 in total

1.  Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse model.

Authors:  Da Sun; Wenyu Sun; Song-Qi Gao; Cheng Wei; Amirreza Naderi; Andrew L Schilb; Josef Scheidt; Sangjoon Lee; Timothy S Kern; Krzysztof Palczewski; Zheng-Rong Lu
Journal:  J Control Release       Date:  2020-12-21       Impact factor: 11.467

2.  Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles.

Authors:  Da Sun; Wenyu Sun; Song-Qi Gao; Jonathan Lehrer; Amirreza Naderi; Cheng Wei; Sangjoon Lee; Andrew L Schilb; Josef Scheidt; Ryan C Hall; Elias I Traboulsi; Krzysztof Palczewski; Zheng-Rong Lu
Journal:  Mol Ther Nucleic Acids       Date:  2022-08-24       Impact factor: 10.183

Review 3.  Directing the Way-Receptor and Chemical Targeting Strategies for Nucleic Acid Delivery.

Authors:  Ricarda Carolin Steffens; Ernst Wagner
Journal:  Pharm Res       Date:  2022-09-15       Impact factor: 4.580
  3 in total

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