Zobrazeno 1 - 10
of 15
pro vyhledávání: '"Liching Cao"'
Autor:
Yonghua Pan, Michelle Rohde, Jennifer Zeitler, Sai Valli Srujana Namburi, Liching Cao, Jing Hu, Kathleen Meyer, Yanmei Lu
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101126- (2023)
Pre-existing antibodies to viral capsids may have a negative impact on the efficacy and safety of adeno-associated virus (AAV)-based gene therapies. Total antibody (TAb) and/or cell-based transduction inhibition (TI) assays have been used to exclude
Externí odkaz:
https://doaj.org/article/e9d56267a88741fab95ee68ffe30680f
Autor:
Makiko Yasuda, Marshall W. Huston, Silvere Pagant, Lin Gan, Susan St. Martin, Scott Sproul, Daniel Richards, Stephen Ballaron, Khaled Hettini, Annemarie Ledeboer, Lillian Falese, Liching Cao, Yanmei Lu, Michael C. Holmes, Kathleen Meyer, Robert J. Desnick, Thomas Wechsler
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 607-619 (2020)
Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the alpha-galactosidase A (GLA) gene, which encodes the exogalactosyl hydrolase, alpha-galactosidase A (α-Gal A). Deficient α-Gal A activity results in the progressive,
Externí odkaz:
https://doaj.org/article/3a1b2560e0e74bff987ed750eaa6a03d
Publikováno v:
Gene Therapy. 30:150-159
Recombinant adeno-associated virus (AAV) vectors are the leading platform for gene delivery for a variety of clinical applications. Patients with preexisting antibodies to AAV are currently excluded from most AAV gene therapy trials to avoid vector n
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4448d3a05ef6cb1d1f98bb1f22113e74
https://doi.org/10.1038/s41434-022-00353-2
https://doi.org/10.1038/s41434-022-00353-2
Autor:
Darshana Jani, Robin Marsden, Michele Gunsior, Laura Schild Hay, Bethany Ward, Kyra J. Cowan, Mitra Azadeh, Breann Barker, Liching Cao, Kristin R. Closson, Kelly Coble, Sanjay L. Dholakiya, Julie Dusseault, Amanda Hays, Carina Herl, Michael E. Hodsdon, Susan C. Irvin, Susan Kirshner, Gerry Kolaitis, Nadia Kulagina, Seema Kumar, Ching Ha Lai, Francesco Lipari, Susana Liu, Keith D. Merdek, Ioana R. Moldovan, Reza Mozaffari, Luying Pan, Corina Place, Veerle Snoeck, Marta Starcevic Manning, Dennis Stocker, Magdalena Tary-Lehmann, Amy Turner, Inna Vainshtein, Daniela Verthelyi, William T. Williams, Haoheng Yan, Weili Yan, Lili Yang, Lin Yang, Jennifer Zemo, Zhandong Don Zhong
Publikováno v:
The AAPS journalReferences. 24(6)
A clear scientific and operational need exists for harmonized bioanalytical immunogenicity study reporting to facilitate communication of immunogenicity findings and expedient review by industry and health authorities. To address these key bioanalyti
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b6785f559d1c0afdbcb7a3a0839baf45
https://pubmed.ncbi.nlm.nih.gov/36307592
https://pubmed.ncbi.nlm.nih.gov/36307592
Autor:
Paul Harmatz, Carlos E. Prada, Barbara K. Burton, Heather Lau, Craig M. Kessler, Liching Cao, Marina Falaleeva, Andres G. Villegas, Jennifer Zeitler, Kathleen Meyer, Weston Miller, Cheryl Wong Po Foo, Sagar Vaidya, Wendy Swenson, Lisa H. Shiue, Didier Rouy, Joseph Muenzer
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 30(12)
Zinc-finger nuclease (ZFN)-based in vivo genome editing is a novel treatment that can potentially provide lifelong protein replacement with single intravenous administration. Three first-in-human open-label ascending single-dose phase 1/2 studies wer
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::edc1d52aa50c44bdf5871e27d0e74055
https://pubmed.ncbi.nlm.nih.gov/36299240
https://pubmed.ncbi.nlm.nih.gov/36299240
Autor:
Daniel Richards, Thomas Wechsler, Khaled Hettini, Lillian Falese, Lin Gan, Michael C. Holmes, Robert J. Desnick, Annemarie Ledeboer, Makiko Yasuda, Kathleen Meyer, Silvere Pagant, Liching Cao, Stephen Ballaron, Yanmei Lu, Scott Sproul, Marshall W. Huston, Susan St Martin
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 607-619 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the alpha-galactosidase A (GLA) gene, which encodes the exogalactosyl hydrolase, alpha-galactosidase A (α-Gal A). Deficient α-Gal A activity results in the progressive,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dcfeb821b6fd702f6a044ddc55770e72
http://www.sciencedirect.com/science/article/pii/S2329050120301510
http://www.sciencedirect.com/science/article/pii/S2329050120301510
Publikováno v:
An Introduction to Bioanalysis of Biopharmaceuticals ISBN: 9783030971922
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::05fd6355a6a41e2c844dc7ca96e6787b
https://doi.org/10.1007/978-3-030-97193-9_5
https://doi.org/10.1007/978-3-030-97193-9_5
Autor:
Liching Cao
Publikováno v:
AAPS Advances in the Pharmaceutical Sciences Series ISBN: 9783030971922
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::727d9de9f242363264516e74d485f5f6
https://doi.org/10.1007/978-3-030-97193-9
https://doi.org/10.1007/978-3-030-97193-9
Autor:
Robert J. Hopkin, Jaya Ganesh, Patrick Deegan, Ozlem Goker-Alpan, John Bernat, William R. Wilcox, Madeleine V. Pahl, Chester B. Whitley, Derralynn Hughes, Kathleen Nicholls, Liching Cao, Michael Chen, Lisa H. Shiue, Emma Bowden, Sravan Jaggumantri, Cristobal Passalacqua, Bernard Souberbielle, Bettina M. Cockroft
Publikováno v:
Molecular Genetics and Metabolism. 138:107152
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::2a2d91d5c71bb87b4993802f2bffe4a9
https://doi.org/10.1016/j.ymgme.2022.107152
https://doi.org/10.1016/j.ymgme.2022.107152
Publikováno v:
Molecular Genetics and Metabolism. 135:S37
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ca9f496e9124f6f0ec16309a8055c147
https://doi.org/10.1016/j.ymgme.2021.11.081
https://doi.org/10.1016/j.ymgme.2021.11.081