Results 31 to 40 of about 214,719 (303)

Development of a self-silencing adenovirus for the efficient manufacture of adeno-associated virus vectors

, 2021
Adeno-associated virus (AAV) shows great promise for gene therapy, however scalability, yield and quality remain significant issues. The most common ‘helper-free’ method used to produce recombinant AAV (rAAV) for pre-clinical/clinical application is ...
Su, Weiheng
core   +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

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

Familial and genetic overlap between Sjögren’s disease and other autoimmune diseases

Frontiers in Immunology
PurposeTo evaluate familial clustering of Sjögren’s disease (SjD) with other autoimmune diseases and to characterize shared genetic architecture using an integrative genomic approach.MethodsMeta-analysis identified studies assessing autoimmune disease ...
Hitomi Ono-Minagi, Hitomi Ono-Minagi, Takuma Ohnishi, Natalie Atyeo, Kentaro Okuno, Peter D. Burbelo, John A. Chiorini   +6 more
doaj   +1 more source

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice

, 2009
Background: Adeno-associated virus serotype 2 (AAV2) vectors show considerable promise for ocular gene transfer. However, one potential barrier to efficacious long-term therapy is the development of immune responses against the vector or transgene ...
Ali, R.R., Buch, P, Barker, S.E., Robbie, S.J., Natkunarajah, M, Maclaren, Robert, Broderick, CA, Smith, A.J., Natkunarajah, M., Duran, Y, Buch, P., Ali, RR, Maclaren, RE, Balaggan, K.S., Duran, Y., MacLaren, R.E., Bainbridge, J.W. B., Bainbridge, JW, Barker, SE, Broderick, C.A., Robbie, SJ, Balaggan, KS, Smith, AJ   +22 more
core   +1 more source

Viral Hybrid Vectors for Somatic Integration - Are They the Better Solution? [PDF]

, 2009
The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based
Anja Ehrhardt, Nadine Müther, Ehrhardt, Anja, Nadja Noske, Müther, Nadine, Noske, Nadja, Nadine Müther   +6 more
core   +1 more source

Image_1_Co-transduction of dual-adeno-associated virus vectors in the neonatal and adult mouse utricles.TIF

, 2022
Adeno-associated virus (AAV)-mediated gene transfer is an efficient method of gene over-expression in the vestibular end organs. However, AAV has limited usefulness for delivering a large gene, or multiple genes, due to its small packaging capacity ...
Jing-Ying Guo (743732), Zhong-Rui Chen (11848124), Jun-Yi Xu (13980396), Shu-Sheng Gong (743733), Zi-Jing Yang (13980399), Ke Liu (121889), Wei Su (89824), Shan Liu (193759), Lu He (811687), Guo-Peng Wang (743731)   +9 more
core   +1 more source

Biosafety of Recombinant Adeno-associated Virus Vectors [PDF]

Current Gene Therapy, 2014
It is hoped that the use of gene transfer technology to treat both monogenetic and acquired diseases may soon become a common therapy option in medicine. For gene therapy to achieve this objective, any gene delivery method will have to meet several criteria, including ease of manufacturing, efficient gene transfer to target tissue, long-term gene ...
Dismuke, D.J., Tenenbaum, L., Samulski, R.J.   +2 more
openaire   +3 more sources

Rab14 regulates the transport of human papillomavirus to the trans‐Golgi network for infectious cell entry

FEBS Letters, EarlyView.
This study reveals that the small GTPase Rab14 is necessary for human papillomavirus (HPV) infection and plays an essential role in the transport of virions to the trans‐Golgi network (TGN). HPV in the early endosome (EE), which harbors GTP‐bound Rab14, is transported to the TGN through the switch of Rab14 from its GTP‐bound to GDP‐bound form.
Yoshiyuki Ishii, Iwao Kukimoto
wiley   +1 more source

Adeno-associated virus: from defective virus to effective vector

Virology Journal, 2005
The initial discovery of adeno-associated virus (AAV) mixed with adenovirus particles was not a fortuitous one but rather an expression of AAV biology.
Gonçalves Manuel AFV
doaj   +1 more source