Results 21 to 30 of about 1,108,606 (318)

Gene Therapy

open access: yesHematology/Oncology Clinics of North America, 2017
Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency ...
Daniel E, Bauer, Donald B, Kohn
openaire   +4 more sources

Human Cytomegalovirus Induces Cellular and Humoral Virus-specific Immune Responses in Humanized BLT Mice

open access: yesScientific Reports, 2017
The strict species specificity of Human Cytomegalovirus (HCMV) has impeded our understanding of antiviral adaptive immune responses in the context of a human immune system.
Lindsey B. Crawford   +7 more
doaj   +1 more source

Functional roles of the membrane-associated AAV protein MAAP

open access: yesScientific Reports, 2021
With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved over-lapping genes to maximise the usage of its genome. An example is the recently found ORF in the cap gene, encoding membrane-associated accessory protein (MAAP),
Lionel Galibert   +13 more
doaj   +1 more source

Alphaviruses in Gene Therapy [PDF]

open access: yesViruses, 2009
Alphaviruses are enveloped single stranded RNA viruses, which as gene therapy vectors provide high-level transient gene expression. Semliki Forest virus (SFV), Sindbis virus (SIN) and Venezuelan Equine Encephalitis (VEE) virus have been engineered as efficient replication-deficient and -competent expression vectors.
openaire   +7 more sources

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

Graph Frequency Features of Cancer Gene Co-Expression Networks [PDF]

open access: yesarXiv, 2023
Complex gene interactions play a significant role in cancer progression, driving cellular behaviors that contribute to tumor growth, invasion, and metastasis. Gene co-expression networks model the functional connectivity between genes under various biological conditions.
arxiv  

Gene therapy

open access: yesBiology of Blood and Marrow Transplantation, 1999
For the past two decades, concerted efforts have been made to treat human disease by replacing nonfunctioning genes in cells or by correcting mutations that produce disease. Successful application of these methods could lead to effective therapies for a variety of genetic and acquired diseases, many of which are not treatable today.
openaire   +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

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  

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