Results 41 to 50 of about 396,665 (375)
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
New England Journal of Medicine, 2020 Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of adeno-associated virus encoding a microRNA targeting SOD1.
C. Mueller +15 more
semanticscholar +1 more source
Adeno-associated virus: fit to serve [PDF]
Current Opinion in Virology, 2014 Adeno-associated virus (AAV) is a helper-dependent parvovirus which has not been linked with human disease. This aspect, in combination with its broad cell and tissue tropism, and limited viral host response has made it an attractive vector system for gene therapy.
Eric Zinn, Luk H Vandenberghe
openaire +2 more sources
Adeno-Associated Virus Vectors and Hematology [PDF]
Blood, 1999 Gene therapists have a special fondness for hematology. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other ...
Mark A. Kay, David W. Russell
openaire +3 more sources
Adeno-associated virus capsid assembly is divergent and stochastic
Nature Communications, 2020 Adeno-associated viruses (AAVs) are increasingly used as gene therapy vectors. AAVs package their genome in a non-enveloped T = 1 icosahedral capsid of ~3.8 megaDalton, consisting of 60 subunits of 3 distinct viral proteins (VPs), which vary only in ...
Tobias P. Wörner +7 more
semanticscholar +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
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
BioDrugs, 2017 There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells ...
M. Naso +3 more
semanticscholar +1 more source
AAVR-Displaying Interfaces: Serotype-Independent Adeno-Associated Virus Capture and Local Delivery Systems. [PDF]
, 2019 Interfacing gene delivery vehicles with biomaterials has the potential to play a key role in diversifying gene transfer capabilities, including localized, patterned, and controlled delivery. However, strategies for modifying biomaterials to interact with
Cho, Mira +5 more
core +1 more source
The Interplay between Adeno-Associated Virus and Its Helper Viruses
Viruses, 2020 The adeno-associated virus (AAV) is a small, nonpathogenic parvovirus, which depends on helper factors to replicate. Those helper factors can be provided by coinfecting helper viruses such as adenoviruses, herpesviruses, or papillomaviruses.
A. Meier, C. Fraefel, M. Seyffert
semanticscholar +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 +6 more
core +1 more source
The landscape of viral associations in human cancers [PDF]
, 2020 Here, as part of the Pan-Cancer Analysis of Whole Genomes (PCAWG) Consortium, for which whole-genome and—for a subset—whole-transcriptome sequencing data from 2,658 cancers across 38 tumor types was aggregated, we systematically investigated potential ...
Alawi, Malik +14 more
core +3 more sources