Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle

ανά Schnepp, Bruce C., Chulay, Jeffrey D., Ye, Guo-Jie, Flotte, Terence R., Trapnell, Bruce C., Johnson, Philip R.
Τόπος έκδοσης Hum Gene Ther (2016)

Cellular and Humoral Immune Responses to Alphavirus Replicon Vaccines Expressing Cytomegalovirus pp65, IE1, and gB Proteins

ανά Reap, Elizabeth A., Dryga, Sergey A., Morris, John, Rivers, Bryan, Norberg, Pamela K., Olmsted, Robert A., Chulay, Jeffrey D.
Έκδοση 2007

Preclinical Evaluation of a Recombinant Adeno-Associated Virus Vector Expressing Human Alpha-1 Antitrypsin Made Using a Recombinant Herpes Simplex Virus Production Method

ανά Chulay, Jeffrey D., Ye, Guo-Jie, Thomas, Darby L., Knop, David R., Benson, Janet M., Hutt, Julie A., Wang, Gensheng, Humphries, Margaret, Flotte, Terence R.
Έκδοση 2011

Development and preclinical evaluation of an alphavirus replicon particle vaccine for cytomegalovirus

ανά Reap, Elizabeth A., Morris, John, Dryga, Sergey A., Maughan, Maureen, Talarico, Todd, Esch, Robert E., Negri, Sarah, Burnett, Bruce, Graham, Andrew, Olmsted, Robert A., Chulay, Jeffrey D.
Έκδοση 2007

Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

ανά Ye, Guo-Jie, Budzynski, Ewa, Sonnentag, Peter, Nork, T. Michael, Sheibani, Nader, Gurel, Zafer, Boye, Sanford L., Peterson, James J., Boye, Shannon E., Hauswirth, William W., Chulay, Jeffrey D.
Τόπος έκδοσης Hum Gene Ther (2016)

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin

ανά Ye, Guo-Jie, Budzynski, Ewa, Sonnentag, Peter, Miller, Paul E., Sharma, Alok K., Ver Hoeve, James N., Howard, Kellie, Knop, David R., Neuringer, Martha, McGill, Trevor, Stoddard, Jonathan, Chulay, Jeffrey D.
Τόπος έκδοσης Hum Gene Ther Clin Dev (2015)

Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs

ανά Ye, Guo-jie, Komáromy, András M., Zeiss, Caroline, Calcedo, Roberto, Harman, Christine D., Koehl, Kristin L., Stewart, Gabriel A., Iwabe, Simone, Chiodo, Vince A., Hauswirth, William W., Aguirre, Gustavo D., Chulay, Jeffrey D.
Τόπος έκδοσης Hum Gene Ther Clin Dev (2017)

Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

ανά Gruntman, Alisha M., Gernoux, Gwladys, Tang, Qiushi, Ye, Guo-Jie, Knop, Dave R., Wang, Gensheng, Benson, Janet, Coleman, Kristen E., Keeler, Allison M., Mueller, Christian, Chicoine, Louis G., Chulay, Jeffrey D., Flotte, Terence R.
Τόπος έκδοσης Mol Ther Methods Clin Dev (2019)

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

ανά Brantly, Mark L., Chulay, Jeffrey D., Wang, Lili, Mueller, Christian, Humphries, Margaret, Spencer, L. Terry, Rouhani, Farshid, Conlon, Thomas J., Calcedo, Roberto, Betts, Michael R., Spencer, Carolyn, Byrne, Barry J., Wilson, James M., Flotte, Terence R.
Έκδοση 2009

Prospective Evaluation of Patients With X-Linked Retinoschisis During 18 Months

ανά Pennesi, Mark E., Birch, David G., Jayasundera, K. Thiran, Parker, Maria, Tan, Ou, Gurses-Ozden, Rabia, Reichley, Carrie, Beasley, Kathleen N., Yang, Paul, Weleber, Richard G., Bennett, Lea D., Heckenlively, John R., Kothapalli, Kalyani, Chulay, Jeffrey D.
Τόπος έκδοσης Invest Ophthalmol Vis Sci (2018)

Development and preclinical evaluation of an alphavirus replicon vaccine for influenza

ανά Hubby, Bolyn, Talarico, Todd, Maughan, Maureen, Reap, Elizabeth A., Berglund, Peter, Kamrud, Kurt I., Copp, Laura, Lewis, Whitney, Cecil, Chad, Norberg, Pamela, Wagner, Jordan, Watson, Aubrey, Negri, Sarah, Burnett, Bruce K., Graham, Andrew, Smith, Jonathan F., Chulay, Jeffrey D.
Έκδοση 2007

Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

ανά Song, Chunjuan, Dufour, Valérie L., Cideciyan, Artur V., Ye, Guo-Jie, Swider, Malgorzata, Newmark, Judith A., Timmers, Adrian M., Robinson, Paulette M., Knop, David R., Chulay, Jeffrey D., Jacobson, Samuel G., Aguirre, Gustavo D., Beltran, William A., Shearman, Mark S.
Τόπος έκδοσης Hum Gene Ther (2020)

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia

ανά Ye, Guo-jie, Budzynski, Ewa, Sonnentag, Peter, Nork, T. Michael, Miller, Paul E., Sharma, Alok K., Ver Hoeve, James N., Smith, Leia M., Arndt, Tara, Calcedo, Roberto, Gaskin, Chantelle, Robinson, Paulette M., Knop, David R., Hauswirth, William W., Chulay, Jeffrey D.
Τόπος έκδοσης Hum Gene Ther Clin Dev (2016)

Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia

ανά Ye, Guo-jie, Budzynski, Ewa, Sonnentag, Peter, Nork, T. Michael, Miller, Paul E., McPherson, Leslie, Ver Hoeve, James N., Smith, Leia M., Arndt, Tara, Mandapati, Savitri, Robinson, Paulette M., Calcedo, Roberto, Knop, David R., Hauswirth, William W., Chulay, Jeffrey D.
Τόπος έκδοσης Hum Gene Ther Clin Dev (2016)

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

ανά Mueller, Christian, Gernoux, Gwladys, Gruntman, Alisha M., Borel, Florie, Reeves, Emer P., Calcedo, Roberto, Rouhani, Farshid N., Yachnis, Anthony, Humphries, Margaret, Campbell-Thompson, Martha, Messina, Louis, Chulay, Jeffrey D., Trapnell, Bruce, Wilson, James M., McElvaney, Noel G., Flotte, Terence R.
Τόπος έκδοσης Mol Ther (2017)

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α(1)-Antitrypsin: Interim Results

ανά Flotte, Terence R., Trapnell, Bruce C., Humphries, Margaret, Carey, Brenna, Calcedo, Roberto, Rouhani, Farshid, Campbell-Thompson, Martha, Yachnis, Anthony T., Sandhaus, Robert A., McElvaney, Noel G., Mueller, Christian, Messina, Louis M., Wilson, James M., Brantly, Mark, Knop, David R., Ye, Guo-jie, Chulay, Jeffrey D.
Έκδοση 2011

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

ανά Song, Chunjuan, Conlon, Thomas J, Deng, Wen-Tao, Coleman, Kirsten E., Zhu, Ping, Plummer, Cayrn, Mandapati, Savitri, Hoosear, Mailin Van, Green, Kari B., Sonnentag, Peter, Sharma, Alok K., Timmers, Adrian, Robinson, Paulette M., Knop, David R., Hauswirth, William W., Chulay, Jeffrey D., Shearman, Mark S., Ye, Guo-jie
Τόπος έκδοσης Hum Gene Ther Clin Dev (2018)

Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigme...

ανά Dufour, Valérie L., Cideciyan, Artur V., Ye, Guo-jie, Song, Chunjuan, Timmers, Adrian, Habecker, Perry L., Pan, Wei, Weinstein, Nicole M., Swider, Malgorzata, Durham, Amy C., Ying, Gui-Shuang, Robinson, Paulette M., Jacobson, Samuel G., Knop, David R., Chulay, Jeffrey D., Shearman, Mark S., Aguirre, Gustavo D., Beltran, William A.
Τόπος έκδοσης Hum Gene Ther (2020)

REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA

ανά Langlo, Christopher S., Erker, Laura R., Parker, Maria, Patterson, Emily J., Higgins, Brian P., Summerfelt, Phyllis, Razeen, Moataz M., Collison, Frederick T., Fishman, Gerald A., Kay, Christine N., Zhang, Jing, Weleber, Richard G., Yang, Paul, Pennesi, Mark E., Lam, Byron L., Chulay, Jeffrey D., Dubra, Alfredo, Hauswirth, William W., Wilson, David J., Carroll, Joseph
Τόπος έκδοσης Retina (2017)

Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia

ανά Langlo, Christopher S., Patterson, Emily J., Higgins, Brian P., Summerfelt, Phyllis, Razeen, Moataz M., Erker, Laura R., Parker, Maria, Collison, Frederick T., Fishman, Gerald A., Kay, Christine N., Zhang, Jing, Weleber, Richard G., Yang, Paul, Wilson, David J., Pennesi, Mark E., Lam, Byron L., Chiang, John, Chulay, Jeffrey D., Dubra, Alfredo, Hauswirth, William W., Carroll, Joseph
Τόπος έκδοσης Invest Ophthalmol Vis Sci (2016)