Results 41 to 50 of about 1,824,389 (266)

Comparison of two lab-scale protocols for enhanced mRNA-based CAR-T cell generation and functionality

open access: yesScientific Reports, 2023
Process development for transferring lab-scale research workflows to automated manufacturing procedures is critical for chimeric antigen receptor (CAR)-T cell therapies.
Nadine von Auw   +10 more
doaj   +1 more source

Efficient transduction of primary vascular cells by the rare adenovirus serotype 49 vector [PDF]

open access: yes, 2015
Neointima formation and vascular remodelling through vascular smooth muscle cell migration and proliferation can limit the long term success of coronary interventions, for example in coronary artery bypass grafting (CABG).
Baker, Andrew H.   +4 more
core   +2 more sources

Gene-level pharmacogenetic analysis on survival outcomes using gene-trait similarity regression [PDF]

open access: yesAnnals of Applied Statistics 2014, Vol. 8, No. 2, 1232-1255, 2014
Gene/pathway-based methods are drawing significant attention due to their usefulness in detecting rare and common variants that affect disease susceptibility. The biological mechanism of drug responses indicates that a gene-based analysis has even greater potential in pharmacogenetics.
arxiv   +1 more source

Stem-cell-based gene therapy for HIV infection. [PDF]

open access: yes, 2013
Despite the enormous success of combined anti-retroviral therapy, HIV infection is still a lifelong disease and continues to spread rapidly worldwide. There is a pressing need to develop a treatment that will cure HIV infection.
Kitchen, Scott, Zhen, Anjie
core   +2 more sources

Cochlear Gene Therapy [PDF]

open access: yesCold Spring Harbor Perspectives in Medicine, 2012
Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss.
Omar Akil, Lawrence R. Lustig
openaire   +6 more sources

Challenges in constructing genetic instruments for pharmacologic therapies [PDF]

open access: yesarXiv, 2020
The genes that encode the targets of most therapies do not have rare variants with large-effect or common variants with moderate effects on the biomarker reflecting the pharmacologic action of the corresponding therapy. Therefore, providing genetic target validation for most therapies is challenging.
arxiv  

On dynamic network entropy in cancer [PDF]

open access: yesSci Rep. 2012;2:802, 2012
The cellular phenotype is described by a complex network of molecular interactions. Elucidating network properties that distinguish disease from the healthy cellular state is therefore of critical importance for gaining systems-level insights into disease mechanisms and ultimately for developing improved therapies.
arxiv   +1 more source

Idiotype vaccines produced with a non-cytopathic alphavirus self-amplifying RNA vector induce antitumor responses in a murine model of B-cell lymphoma

open access: yesScientific Reports, 2021
A promising therapy for patients with B-cell lymphoma is based on vaccination with idiotype monoclonal antibodies (mAbs). Since idiotypes are different in each tumor, a personalized vaccine has to be produced for each patient.
Erkuden Casales   +11 more
doaj   +1 more source

Circumventing antivector immunity: potential use of nonhuman adenoviral vectors [PDF]

open access: yes, 2014
Adenoviruses are efficient gene delivery vectors based on their ability to transduce a wide variety of cell types and drive high-level transient transgene expression. While there have been advances in modifying human adenoviral (HAdV) vectors to increase
Beard C.W.   +30 more
core   +3 more sources

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

open access: yesEMBO Molecular Medicine
Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), however, adverse ...
Qing Xie   +16 more
doaj   +1 more source

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