Zobrazeno 1 - 10
of 56
pro vyhledávání: '"Guo-Jie Ye"'
Autor:
Alisha M. Gruntman, Gwladys Gernoux, Qiushi Tang, Guo-Jie Ye, Dave R. Knop, Gensheng Wang, Janet Benson, Kristen E. Coleman, Allison M. Keeler, Christian Mueller, Louis G. Chicoine, Jeffrey D. Chulay, Terence R. Flotte
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 233-242 (2019)
Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of serum alpha-1 antitrypsin (AAT) deficiency have shown long-term (5-year) stable transgene ex
Externí odkaz:
https://doaj.org/article/ece0ba0412444a99888e9c8c1783ad84
Autor:
Guo-jie Ye, Chunjuan Song, Judith A Newmark, Artur V. Cideciyan, Paulette M. Robinson, William A. Beltran, Adrian M. Timmers, David R. Knop, Jeffrey D. Chulay, Valerie L. Dufour, Mark S. Shearman, Malgorzata Swider, Samuel G. Jacobson, Gustavo D. Aguirre
Publikováno v:
Human Gene Therapy. 31:743-755
Recombinant adeno-associated viral (rAAV) vector-mediated gene therapy is being developed to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase...
Autor:
Qiushi Tang, Gensheng Wang, Christian Mueller, Gwladys Gernoux, Dave R. Knop, Louis G. Chicoine, Terence R. Flotte, Kristen E. Coleman, Allison M. Keeler, Alisha M. Gruntman, Guo-jie Ye, Janet M. Benson, Jeffrey D. Chulay
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss, Pp 233-242 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of serum alpha-1 antitrypsin (AAT) deficiency have shown long-term (5-year) stable transgene ex
Autor:
Savitri Mandapati, Chunjuan Song, Alok K. Sharma, Mailin Van Hoosear, William W. Hauswirth, Guo-jie Ye, Jeffrey D. Chulay, Adrian M. Timmers, Cayrn Plummer, Ping Zhu, David R. Knop, Kari B. Green, Paulette M. Robinson, Peter Sonnentag, Wen-Tao Deng, Mark S. Shearman, Thomas J. Conlon, Kirsten E. Coleman
Publikováno v:
Human Gene Therapy Clinical Development. 29:188-197
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa
Autor:
Chunjuan, Song, Valérie L, Dufour, Artur V, Cideciyan, Guo-Jie, Ye, Malgorzata, Swider, Judith A, Newmark, Adrian M, Timmers, Paulette M, Robinson, David R, Knop, Jeffrey D, Chulay, Samuel G, Jacobson, Gustavo D, Aguirre, William A, Beltran, Mark S, Shearman
Publikováno v:
Hum Gene Ther
Recombinant adeno-associated viral (rAAV) vector-mediated gene therapy is being developed to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. In preparation for a clinical
Autor:
Chunjuan Song, Mark S. Shearman, Guo-jie Ye, Artur V. Cideciyan, Paulette M. Robinson, Gui-Shuang Ying, Gustavo D. Aguirre, Valerie L. Dufour, Malgorzata Swider, Adrian M. Timmers, William A. Beltran, Nicole M. Weinstein, Perry L. Habecker, Wei Pan, David R. Knop, Samuel G. Jacobson, Jeffrey D. Chulay, Amy C. Durham
Publikováno v:
Hum Gene Ther
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated rAAV2tYF-GRK1-hRPGRco, to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retiniti
Autor:
Chantelle Gaskin, Tanaz Farivar, Heikki T Turunen, David R. Knop, Chunjuan Song, Judith A Newmark, Mark S. Shearman, Steven Pennock, Guo-jie Ye, Adrian M. Timmers, Jilin Liu
Publikováno v:
Human gene therapy. 31(1-2)
Both subretinal dosing and intravitreal (IVT) dosing of adeno-associated virus (AAV) in higher species induce mild and transient inflammatory responses that increase with dose. Foreign protein and foreign DNA are known inducers of inflammation, which
Publikováno v:
Human gene therapy. Clinical development. 30(1)
Autor:
Terence R. Flotte, Guo-jie Ye, Jeffrey D. Chulay, Bruce C. Schnepp, Philip R. Johnson, Bruce C. Trapnell
Publikováno v:
Human Gene Therapy. 27:32-42
Gene augmentation therapy as a strategy to treat alpha-1 antitrypsin (AAT) deficiency has reached phase 2 clinical testing in humans. Sustained serum levels of AAT have been observed beyond one year after intramuscular administration of a recombinant
Autor:
Jeffrey D. Chulay, Jim Peterson, Guo-jie Ye, William W. Hauswirth, Nader Sheibani, Shannon E. Boye, Sanford L. Boye, Ewa Budzynski, Zafer Gurel, T Michael Nork, Peter Sonnentag
Publikováno v:
Human Gene Therapy. 27:72-82
Adeno-associated viral (AAV) vectors containing cone-specific promoters have rescued cone photoreceptor function in mouse and dog models of achromatopsia, but cone-specific promoters have not been optimized for use in primates. Using AAV vectors admi