Literature DB >> 17249581

Lentiviral vectors containing a retinal pigment epithelium specific promoter for leber congenital amaurosis gene therapy. Lentiviral gene therapy for LCA.

Alexis-Pierre Bemelmans1, Corinne Kostic, Dana Hornfeld, Muriel Jaquet, Sylvain V Crippa, William W Hauswirth, Janis Lem, Zhongyan Wang, Daniel E Schorderet, Francis L Munier, Andreas Wenzel, Yvan Arsenijevic.   

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Year:  2006        PMID： 17249581     DOI： 10.1007/0-387-32442-9_35

Source DB:  PubMed          Journal:  Adv Exp Med Biol        ISSN： 0065-2598            Impact factor:   2.622


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  6 in total

Review 1.  Gene therapy for Leber congenital amaurosis: advances and future directions.

Authors:  Robert B Hufnagel; Zubair M Ahmed; Zélia M Corrêa; Robert A Sisk
Journal:  Graefes Arch Clin Exp Ophthalmol       Date:  2012-05-29       Impact factor: 3.117

2.  Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Authors:  Stephen Russell; Jean Bennett; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Sarah McCague; Dominique Cross; Kathleen A Marshall; Jean Walshire; Taylor L Kehoe; Hannah Reichert; Maria Davis; Leslie Raffini; Lindsey A George; F Parker Hudson; Laura Dingfield; Xiaosong Zhu; Julia A Haller; Elliott H Sohn; Vinit B Mahajan; Wanda Pfeifer; Michelle Weckmann; Chris Johnson; Dina Gewaily; Arlene Drack; Edwin Stone; Katie Wachtel; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2017-07-14       Impact factor: 79.321

Review 3.  A comprehensive review of retinal gene therapy.

Authors:  Shannon E Boye; Sanford L Boye; Alfred S Lewin; William W Hauswirth
Journal:  Mol Ther       Date:  2013-01-29       Impact factor: 11.454

4.  Therapeutic Benefits from Nanoparticles: The Potential Significance of Nanoscience in Retinal Degenerative Diseases.

Authors:  Raju V S Rajala
Journal:  J Mol Biol Ther       Date:  2019

Review 5.  Lipid Nanoparticles for Ocular Gene Delivery.

Authors:  Yuhong Wang; Ammaji Rajala; Raju V S Rajala
Journal:  J Funct Biomater       Date:  2015-06-08

6.  Nanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo.

Authors:  Ammaji Rajala; Yuhong Wang; Ye Zhu; Michelle Ranjo-Bishop; Jian-Xing Ma; Chuanbin Mao; Raju V S Rajala
Journal:  Nano Lett       Date:  2014-08-12       Impact factor: 11.189
  6 in total

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