Results 21 to 30 of about 9,435,635 (153)

Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]

, 2008
Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Amit Nathwani, Baker, AH, Lorraine M Work, Tracey Graham, McIntosh, JB, Jenny McIntosh, Nathwani, A, Work, LM, Graham, T, McIntosh, J, Andrew H Baker   +10 more
core   +1 more source

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

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

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

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Ward, N.J., Buckley, S.M.K., Ward, NJ, Chan, J.K.Y., Waddington, S.N., Rahim, A.A., Waddington, SN, Rahim, AA, Chan, JK, Mckay, T.R., Howe, S.J., Howe, SJ, McKay, TR, Natalie J. Ward, Ahad A. Rahim, Steven J. Howe, Jerry K.Y.Chan, Suzanne M.K. Buckley, Simon N. Waddington, Tristan R. McKay   +20 more
core   +1 more source

Herpes simplex virus co-infection facilitates rolling circle replication of the adeno-associated virus genome. [PDF]

, 2021
Adeno-associated virus (AAV) genome replication only occurs in the presence of a co-infecting helper virus such as adenovirus type 5 (AdV5) or herpes simplex virus type 1 (HSV-1).
Anouk Lkharrazi, Cornel Fraefel, Ros, Carlos, Fraefel, Cornel, Remo Leisi, Anita Felicitas Meier, Carlos Ros, Lkharrazi, Anouk, Tobler, Kurt, Leisi, Remo, Kurt Tobler, Meier, Anita Felicitas, Meier, Anita Felicitas; https://orcid.org/, Tobler, Kurt; https://orcid.org/, Leisi, Remo; https://orcid.org/, Fraefel, Cornel; https://orcid.org/, Ros, Carlos; https://orcid.org/, Lkharrazi, Anouk; https://orcid.org/   +17 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

Mass spectrometry friendly pH-gradient anion exchange chromatography for separation of full and empty Adeno-Associated Virus (AAV) capsids [PDF]

, 2023
The proportion of full and empty capsids represents a critical quality attribute of adeno-associated virus (AAV)-based therapeutics. In this study, pH-gradient anion exchange chromatography was utilized for the separation of full and empty capsid species.
Florian, Füssl, Felipe, Guapo, Lisa, Strasser, Jonathan, Bones   +3 more
core   +2 more sources

Generation of enhanced gene delivery vectors by directed evolution of adeno-associated virus [PDF]

, 2006
Despite promising advance in the development of viral vectors based on AAV for human gene therapy, several major hurdles for a more general use remain.
Endell, Jan
core   +1 more source

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

, 2020
International audienceRecombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer.
Ronzitti, Giuseppe, Mingozzi, Federico, Gross, David-Alexandre   +2 more
core   +1 more source