Literature DB >> 19675341

Vision 1 year after gene therapy for Leber's congenital amaurosis.

Artur V Cideciyan, William W Hauswirth, Tomas S Aleman, Shalesh Kaushal, Sharon B Schwartz, Sanford L Boye, Elizabeth A M Windsor, Thomas J Conlon, Alexander Sumaroka, Alejandro J Roman, Barry J Byrne, Samuel G Jacobson.   

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Year:  2009        PMID： 19675341      PMCID： PMC2847775          DOI： 10.1056/NEJMc0903652

Source DB:  PubMed          Journal:  N Engl J Med        ISSN： 0028-4793            Impact factor:   91.245


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  5 in total

1.  Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.

Authors:  Artur V Cideciyan; Tomas S Aleman; Sanford L Boye; Sharon B Schwartz; Shalesh Kaushal; Alejandro J Roman; Ji-Jing Pang; Alexander Sumaroka; Elizabeth A M Windsor; James M Wilson; Terence R Flotte; Gerald A Fishman; Elise Heon; Edwin M Stone; Barry J Byrne; Samuel G Jacobson; William W Hauswirth
Journal:  Proc Natl Acad Sci U S A       Date:  2008-09-22       Impact factor: 11.205

2.  Effect of gene therapy on visual function in Leber's congenital amaurosis.

Authors:  James W B Bainbridge; Alexander J Smith; Susie S Barker; Scott Robbie; Robert Henderson; Kamaljit Balaggan; Ananth Viswanathan; Graham E Holder; Andrew Stockman; Nick Tyler; Simon Petersen-Jones; Shomi S Bhattacharya; Adrian J Thrasher; Fred W Fitzke; Barrie J Carter; Gary S Rubin; Anthony T Moore; Robin R Ali
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

3.  Safety and efficacy of gene transfer for Leber's congenital amaurosis.

Authors:  Albert M Maguire; Francesca Simonelli; Eric A Pierce; Edward N Pugh; Federico Mingozzi; Jeannette Bennicelli; Sandro Banfi; Kathleen A Marshall; Francesco Testa; Enrico M Surace; Settimio Rossi; Arkady Lyubarsky; Valder R Arruda; Barbara Konkle; Edwin Stone; Junwei Sun; Jonathan Jacobs; Lou Dell'Osso; Richard Hertle; Jian-xing Ma; T Michael Redmond; Xiaosong Zhu; Bernd Hauck; Olga Zelenaia; Kenneth S Shindler; Maureen G Maguire; J Fraser Wright; Nicholas J Volpe; Jennifer Wellman McDonnell; Alberto Auricchio; Katherine A High; Jean Bennett
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

Review 4.  Leber congenital amaurosis: genes, proteins and disease mechanisms.

Authors:  Anneke I den Hollander; Ronald Roepman; Robert K Koenekoop; Frans P M Cremers
Journal:  Prog Retin Eye Res       Date:  2008-06-01       Impact factor: 21.198

5.  Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.

Authors:  William W Hauswirth; Tomas S Aleman; Shalesh Kaushal; Artur V Cideciyan; Sharon B Schwartz; Lili Wang; Thomas J Conlon; Sanford L Boye; Terence R Flotte; Barry J Byrne; Samuel G Jacobson
Journal:  Hum Gene Ther       Date:  2008-10       Impact factor: 5.695
  5 in total
  107 in total

1.  Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

Authors:  Ian E Alexander; Cindy Kok; Allison P Dane; Sharon C Cunningham
Journal:  J Inherit Metab Dis       Date:  2012-03-09       Impact factor: 4.982

2.  Recombinant adeno-associated viral vector production and purification.

Authors:  Jin-Hong Shin; Yongping Yue; Dongsheng Duan
Journal:  Methods Mol Biol       Date:  2012

3.  Temporal interactions during paired-electrode stimulation in two retinal prosthesis subjects.

Authors:  Alan Horsager; Geoffrey M Boynton; Robert J Greenberg; Ione Fine
Journal:  Invest Ophthalmol Vis Sci       Date:  2011-02-01       Impact factor: 4.799

4.  Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.

Authors:  J Pang; S E Boye; B Lei; S L Boye; D Everhart; R Ryals; Y Umino; B Rohrer; J Alexander; J Li; X Dai; Q Li; B Chang; R Barlow; W W Hauswirth
Journal:  Gene Ther       Date:  2010-03-18       Impact factor: 5.250

Review 5.  Mitochondrial medicine: to a new era of gene therapy for mitochondrial DNA mutations.

Authors:  Hélène Cwerman-Thibault; José-Alain Sahel; Marisol Corral-Debrinski
Journal:  J Inherit Metab Dis       Date:  2010-06-23       Impact factor: 4.982

Review 6.  AAV-mediated gene therapy in mouse models of recessive retinal degeneration.

Authors:  J-J Pang; L Lei; X Dai; W Shi; X Liu; A Dinculescu; J H McDowell
Journal:  Curr Mol Med       Date:  2012-03       Impact factor: 2.222

Review 7.  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

Review 8.  What Is Next for Retinal Gene Therapy?

Authors:  Luk H Vandenberghe
Journal:  Cold Spring Harb Perspect Med       Date:  2015-04-15       Impact factor: 6.915

9.  Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase.

Authors:  Thomas J Conlon; Kirsten Erger; Stacy Porvasnik; Travis Cossette; Cheryl Roberts; Lynn Combee; Saleem Islam; Jeffry Kelley; Denise Cloutier; Nathalie Clément; Corinne R Abernathy; Barry J Byrne
Journal:  Hum Gene Ther Clin Dev       Date:  2013-09       Impact factor: 5.032

10.  Determining consequences of retinal membrane guanylyl cyclase (RetGC1) deficiency in human Leber congenital amaurosis en route to therapy: residual cone-photoreceptor vision correlates with biochemical properties of the mutants.

Authors:  Samuel G Jacobson; Artur V Cideciyan; Igor V Peshenko; Alexander Sumaroka; Elena V Olshevskaya; Lihui Cao; Sharon B Schwartz; Alejandro J Roman; Melani B Olivares; Sam Sadigh; King-Wai Yau; Elise Heon; Edwin M Stone; Alexander M Dizhoor
Journal:  Hum Mol Genet       Date:  2012-10-03       Impact factor: 6.150
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