Long-term Preservation of Cone Photoreceptors and Restoration of Cone Function by Gene Therapy in the Guanylate Cyclase-1 Knockout (GC1KO) Mouse

nork Boye, Sanford L., Conlon, Thomas, Erger, Kirsten, Ryals, Renee, Neeley, Andy, Cossette, Travis, Pang, Jijing, Dyka, Frank M., Hauswirth, William W., Boye, Shannon E.
Argitaratua 2011

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

nork Keeler, Allison M, Conlon, Thomas, Walter, Glenn, Zeng, Huadong, Shaffer, Scott A, Dungtao, Fu, Erger, Kirsten, Cossette, Travis, Tang, Qiushi, Mueller, Christian, Flotte, Terence R
Argitaratua 2012

Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 Human Acid α-Glucosidase

nork Conlon, Thomas J., Erger, Kirsten, Porvasnik, Stacy, Cossette, Travis, Roberts, Cheryl, Combee, Lynn, Islam, Saleem, Kelley, Jeffry, Cloutier, Denise, Clément, Nathalie, Abernathy, Corinne R., Byrne, Barry J.
Argitaratua 2013

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

nork Deng, Wen-Tao, Dyka, Frank M., Dinculescu, Astra, Li, Jie, Zhu, Ping, Chiodo, Vince A., Boye, Sanford L., Conlon, Thomas J., Erger, Kirsten, Cossette, Travis, Hauswirth, William W.
Argitaratua izan da Hum Gene Ther (2015)

Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors

nork Conlon, Thomas J., Mah, Cathryn S., Pacak, Christina A., Rucker Henninger, Mary B., Erger, Kirsten E., Jorgensen, Marda L., Lee, C. Chang I., Tarantal, Alice F., Byrne, Barry J.
Argitaratua izan da Hum Gene Ther Clin Dev (2016)

Reprogramming Adipose Tissue-Derived Mesenchymal Stem Cells into Pluripotent Stem Cells by a Mutant Adeno-Associated Viral Vector

nork Chen, Mong-Jen, Lu, Yuanqing, Hamazaki, Takashi, Tsai, Hsin-Yin, Erger, Kirsten, Conlon, Thomas, Elshikha, Ahmed S., Li, Hong, Srivastava, Arun, Yao, Chunli, Brantly, Mark, Chiodo, Vince, Hauswirth, William, Terada, Naohiro, Song, Sihong
Argitaratua 2014

LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile

nork Koilkonda, Rajeshwari, Yu, Hong, Talla, Venu, Porciatti, Vittorio, Feuer, William J., Hauswirth, William W., Chiodo, Vince, Erger, Kirsten E., Boye, Sanford L., Lewin, Alfred S., Conlon, Thomas J., Renner, Lauren, Neuringer, Martha, Detrisac, Carol, Guy, John
Argitaratua izan da Invest Ophthalmol Vis Sci (2014)

Glycogen Storage Disease Type Ia in Canines: A Model for Human Metabolic and Genetic Liver Disease

nork Specht, Andrew, Fiske, Laurie, Erger, Kirsten, Cossette, Travis, Verstegen, John, Campbell-Thompson, Martha, Struck, Maggie B., Lee, Young Mok, Chou, Janice Y., Byrne, Barry J., Correia, Catherine E., Mah, Cathryn S., Weinstein, David A., Conlon, Thomas J.
Argitaratua 2011

An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery

nork Lee, Ni-Chung, Falk, Darin J, Byrne, Barry J, Conlon, Thomas J, Clement, Nathalie, Porvasnik, Stacy, Jorgensen, Marda L, Potter, Mark, Erger, Kirsten E, Watson, Rachael, Ghivizzani, Steven C, Chiu, Hung-Chuan, Chien, Yin-Hsiu, Hwu, Wuh-Liang
Argitaratua 2012

The Human Rhodopsin Kinase Promoter in an AAV5 Vector Confers Rod- and Cone-Specific Expression in the Primate Retina

nork Boye, Shannon E., Alexander, John J., Boye, Sanford L., Witherspoon, Clark D., Sandefer, Kristen J., Conlon, Thomas J., Erger, Kirsten, Sun, Jingfen, Ryals, Renee, Chiodo, Vince A., Clark, Mark E., Girkin, Christopher A., Hauswirth, William W., Gamlin, Paul D.
Argitaratua 2012

Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa

nork Conlon, Thomas J., Deng, Wen-Tao, Erger, Kirsten, Cossette, Travis, Pang, Ji-jing, Ryals, Renee, Clément, Nathalie, Cleaver, Brian, McDoom, Issam, Boye, Shannon E., Peden, Marc C., Sherwood, Mark B., Abernathy, Corinne R., Alkuraya, Fowzan S., Boye, Sanford L., Hauswirth, William W.
Argitaratua 2013

Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years

nork Jacobson, Samuel G., Cideciyan, Artur V., Ratnakaram, Ramakrishna, Heon, Elise, Schwartz, Sharon B., Roman, Alejandro J., Peden, Marc C., Aleman, Tomas S., Boye, Sanford L., Sumaroka, Alexander, Conlon, Thomas J., Calcedo, Roberto, Pang, Ji-jing, Erger, Kirsten E., Olivares, Melani B., Mullins, Cristina L., Swider, Malgorzata, Kaushal, Shalesh, Feuer, Willam J., Iannaccone, Alessandro, Fishman, Gerald A., Stone, Edwin M., Byrne, Barry J., Hauswirth, William W.
Argitaratua 2011