Gene Therapy

Suwajanakorn, Lane, Go, Hartley, Oxenreiter, Wu, Gragoudas E, Sullivan, Montazeri, Kim I. Impact of gene expression profiling on diagnosis and survival after metastasis in patients with uveal melanoma.

Karg M, Lu YR, Refaian N, Cameron J, Hoffmann E, Hoppe C, Shirahama S, Shah M, Krasniqi D, Krishnan A, Shrestha M, Guo Y, Cermak J, Walthier M, Broniowska K, Rosenzweig-Lipson S, Gregory-Ksander M, Sinclair D, Ksander B. Sustained Vision Recovery by OSK Gene Therapy in a Mouse Model of Glaucoma.

Xue Y, Cepko C. Gene Therapies for Retinitis Pigmentosa that Target Glucose Metabolism.

Ashraf S, Deshpande N, Vasanth S, Melangath G, Wong R, Zhao Y, Price M, Price F, Jurkunas U. Dysregulation of DNA repair genes in Fuchs endothelial corneal dystrophy.

Florea M, Nicolaou F, Pacouret S, Zinn E, Sanmiguel J, Andres-Mateos E, Unzu C, Wagers A, Vandenberghe L. High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography.

Hsu Y, Bhattarai S, Thompson J, Mahoney A, Thomas J, Mayer S, Datta P, Garrison J, Searby C, Vandenberghe L, Seo S, Sheffield V, Drack A. Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model.

Carelli V, Newman N, Yu-Wai-Man P, Biousse V, Moster M, Subramanian P, Vignal-Clermont C, Wang AG, Donahue S, Leroy B, Sergott R, Klopstock T, Sadun A, Fernández GR, Chwalisz B, Banik R, Girmens JF, La Morgia C, DeBusk A, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA, Group S. Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.

Yang JH, Hayano M, Griffin P, Amorim J, Bonkowski M, Apostolides J, Salfati E, Blanchette M, Munding E, Bhakta M, Chew YC, Guo W, Yang X, Maybury-Lewis S, Tian X, Ross J, Coppotelli G, Meer M, Rogers-Hammond R, Vera D, Lu YR, Pippin J, Creswell M, Dou Z, Xu C, Mitchell S, Das A, O’Connell B, Thakur S, Kane A, Su Q, Mohri Y, Nishimura E, Schaevitz L, Garg N, Balta AM, Rego M, Gregory-Ksander M, Jakobs T, Zhong L, Wakimoto H, El Andari J, Grimm D, Mostoslavsky R, Wagers A, Tsubota K, Bonasera S, Palmeira C, Seidman J, Seidman C, Wolf N, Kreiling J, Sedivy J, Murphy G, Green R, Garcia B, Berger S, Oberdoerffer P, Shankland S, Gladyshev VN, Ksander B, Pfenning A, Rajman L, Sinclair D. Loss of epigenetic information as a cause of mammalian aging.

Nagel-Wolfrum K, Fadl B, Becker M, Wunderlich K, Schäfer J, Sturm D, Fritze J, Gür B, Kaplan L, Andreani T, Goldmann T, Brooks M, Starostik M, Lokhande A, Apel M, Fath K, Stingl K, Kohl S, Deangelis M, Schlötzer-Schrehardt U, Kim I, Owen L, Vetter J, Pfeiffer N, Andrade-Navarro M, Grosche A, Swaroop A, Wolfrum U. Expression and subcellular localization of USH1C/harmonin in human retina provides insights into pathomechanisms and therapy.

Katz M, Hadas Y, Bailey R, Fazal S, Vincek A, Madjarova S, Shtraizent N, Vandenberghe L, Eliyahu E. Efficient cardiac gene transfer and early-onset expression of a synthetic adeno-associated viral vector, Anc80L65, after intramyocardial administration.