Zobrazeno 1 - 5
of 5
pro vyhledávání: '"Jennifer Wellman McDonnell"'
Autor:
Arkady Lyubarsky, Jean Bennett, Xiaosong Zhu, Maureen G. Maguire, Eric A. Pierce, Enrico Maria Surace, Kathleen A. Marshall, Barbara A. Konkle, Sandro Banfi, L. F. Dell'Osso, Jeannette L. Bennicelli, Jonathan B. Jacobs, Edwin M. Stone, J. Fraser Wright, Richard W. Hertle, Kenneth S. Shindler, Edward N. Pugh, Nicholas J. Volpe, Jennifer Wellman McDonnell, Katherine A. High, Junwei Sun, Valder R. Arruda, Francesco Testa, Albert M. Maguire, Jian Xing Ma, Alberto Auricchio, Federico Mingozzi, Bernd Hauck, Olga Zelenaia, T. Michael Redmond, Francesca Simonelli, Settimio Rossi
Publikováno v:
New England Journal of Medicine. 358:2240-2248
S um m a r y Leber's congenital amaurosis (LCA) is a group of inherited blinding diseases with onset during childhood. One form of the disease, LCA2, is caused by mutations in the retinal pigment epithelium-specific 65-kDa protein gene (RPE65). We in
Autor:
Pete Smith, Joseph Prado, Glenn F. Pierce, Floro Cataniag, Shangzhen Zhou, Katherine A. High, Alex Tai, Jac Luna, Jennifer Wellman McDonnell, J. Fraser Wright, Guang Qu, Jurg M. Sommer, Jingmin Zhou, Peter Colosi, Bernd Hauck, Jennifer Bahr-Davidson
Publikováno v:
Journal of Virological Methods. 140:183-192
Adeno-associated virus (AAV) empty capsids typically co-purify with genome containing AAV2 vectors purified by column chromatography. This study describes a method to remove empty capsids from genome containing vector particles by anion exchange chro
Autor:
Pete Smith, Michael A. Lochrie, Peter Colosi, Kris Jones, Gwen Tatsuno, Alejandra E. Arbetman, Shang Zhen Zhou, Cheryl Pater, Michiko Kachi, Glenn F. Pierce, Jennifer Wellman McDonnell, Peter A. Campochiaro, Shu Kachi
Publikováno v:
Virology. 353:68-82
Using polymerase chain reactions and genome walking strategies, adeno-associated virus (AAV)-like capsid genes were isolated from rat and mouse liver genomic DNA, where they are present at
Autor:
Glenn F. Pierce, Shangzhen Zhou, Michael A. Lochrie, Jennifer Wellman McDonnell, B Christie, Peter Colosi, Gwen P. Tatsuno, Richard T. Surosky
Publikováno v:
Journal of Virology. 80:821-834
Mutations were made at 64 positions on the external surface of the adeno-associated virus type 2 (AAV-2) capsid in regions expected to bind antibodies. The 127 mutations included 57 single alanine substitutions, 41 single nonalanine substitutions, 27
Autor:
Jeannette L. Bennicelli, Carmela Acerra, Sandro Banfi, Settimio Rossi, Carmela Ziviello, Kenneth S. Shindler, T. Michael Redmond, Federico Mingozzi, Kathleen A. Marshall, Francesca Simonelli, Xiaosong Zhu, Enrico Maria Surace, Eric A. Pierce, Katherine A. High, Junwei Sun, Albert M. Maguire, Jennifer Wellman McDonnell, Francesco Testa, J. Fraser Wright, Alberto Auricchio, Gui-Shuang Ying, Jean Bennett
Publikováno v:
Molecular Therapy
Molecular Therapy; Vol 18
Molecular Therapy; Vol 18
The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subr