Results 21 to 30 of about 110,488 (256)

Process Improvement of Adeno-Associated Virus Production

Frontiers in Chemical Engineering, 2022
Adeno-associated viruses (AAVs) have been well characterized and used to deliver therapeutic genes for diseases treatment in clinics and basic research. This study used the triple transient transfection of AAV-DJ/8 as a model expression system to develop
Jia-Shiung Guan, Kai Chen, Yingnan Si, Taehyun Kim, Zhuoxin Zhou, Seulhee Kim, Lufang Zhou, Xiaoguang Liu   +7 more
doaj   +1 more source

Profiling of Glycan Receptors for Minute Virus of Mice in Permissive Cell Lines Towards Understanding the Mechanism of Cell Recognition [PDF]

, 2013
The recognition of sialic acids by two strains of minute virus of mice (MVM), MVMp (prototype) and MVMi (immunosuppressive), is an essential requirement for successful infection.
Agbandje-McKenna, M, Chen, X, Cotmore, S, Cummings, RD, Dell, A, Halder, S, Haslam, SM, Heimburg-Molinaro, J, McKenna, R, North, SJ, Smith, DF, Tattersall, P, Trollope, AJ   +12 more
core   +1 more source

In vivo genetic manipulation of inner ear connexin expression by bovine adeno-Associated viral vectors [PDF]

, 2017
We have previously shown that in vitro transduction with bovine adeno-associated viral (BAAV) vectors restores connexin expression and rescues gap junction coupling in cochlear organotypic cultures from connexin-deficient mice that are models DFNB1 ...
Campioni, Matteo, Chiorini, John A., Crispino, Giulia, Di Pasquale, Giovanni, Galindo Ramirez, Fabian, Mammano, Fabio, Praetorius, Mark, Zorzi, Veronica   +7 more
core   +1 more source

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]

, 2013
Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S, Boye, SE, Chiodo, V, Dyka, F, Fofo, H, Hauswirth, WW, Lopes, VS, Louie, CM, Williams, DS   +8 more
core   +1 more source

Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease

Blood Advances, 2017
: Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction.
Randall K. Merling, Douglas B. Kuhns, Colin L. Sweeney, Xiaolin Wu, Sandra Burkett, Jessica Chu, Janet Lee, Sherry Koontz, Giovanni Di Pasquale, Sandra A. Afione, John A. Chiorini, Elizabeth M. Kang, Uimook Choi, Suk See De Ravin, Harry L. Malech   +14 more
doaj   +1 more source

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Molecular Therapy: Methods & Clinical Development, 2021
Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated
John W. Day, Richard S. Finkel, Eugenio Mercuri, Kathryn J. Swoboda, Melissa Menier, Rudolf van Olden, Sitra Tauscher-Wisniewski, Jerry R. Mendell   +7 more
doaj   +1 more source

Adenovirus infection in pediatric liver transplant recipients [PDF]

, 1992
A retrospective review of adenoviral infection in pediatric liver transplant recipients was done at Children’s Hospital of Pittsburgh to define its epidemiology and clinical importance.
Green, M, Michaels, MG, Starzl, TE, Wald, ER   +3 more
core   +2 more sources

The natural dietary genistein boosts bacteriophage-mediated cancer cell killing by improving phage-targeted tumor cell transduction [PDF]

, 2016
Gene therapy has long been regarded as a promising treatment for cancer. However, cancer gene therapy is still facing the challenge of targeting gene delivery vectors specifically to tumors when administered via clinically acceptable non-invasive ...
Albahrani, M, Bentayebi, K, Hajitou, A, Przystal, J, Suwan, K, Tsafa, E   +5 more
core   +2 more sources

Gene Transfer in Rodent Nervous Tissue Following Hindlimb Intramuscular Delivery of Recombinant Adeno-Associated Virus Serotypes AAV2/6, AAV2/8, and AAV2/9

Neuroscience Insights, 2019
Recombinant adeno-associated virus (rAAV) vectors have emerged as the safe vehicles of choice for long-term gene transfer in mammalian nervous system. Recombinant adeno-associated virus–mediated localized gene transfer in adult nervous system following ...
Asad Jan, Mette Richner, Christian B Vægter, Jens R Nyengaard, Poul H Jensen   +4 more
doaj   +1 more source

Status and Prospect of Gene Therapy for Hemophilia

罕见病研究, 2022
Hemophilia is a congenital hemorrhagic disease caused by genetic abnormalities in coagulation factor Ⅷ or factor Ⅸ. Current conventional therapy to prevent bleeding requires frequent intravenous injections of coagulation factor concentrates from early ...
DAI Xinyue, ZHANG Lei
doaj   +1 more source