Results 21 to 30 of about 1,173,119 (313)

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

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

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

Insights into PI3K/AKT signaling in B cell development and chronic lymphocytic leukemia

open access: yesFEBS Letters, EarlyView.
This Review explores how the phosphoinositide 3‐kinase and protein kinase B pathway shapes B cell development and drives chronic lymphocytic leukemia, a common blood cancer. It examines how signaling levels affect disease progression, addresses treatment challenges, and introduces novel experimental strategies to improve therapies and patient outcomes.
Maike Buchner
wiley   +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

The cytoskeletal control of B cell receptor and integrin signaling in normal B cells and chronic lymphocytic leukemia

open access: yesFEBS Letters, EarlyView.
In lymphoid organs, antigen recognition and B cell receptor signaling rely on integrins and the cytoskeleton. Integrins act as mechanoreceptors, couple B cell receptor activation to cytoskeletal remodeling, and support immune synapse formation as well as antigen extraction.
Abhishek Pethe, Tanja Nicole Hartmann
wiley   +1 more source

Macrophage Transcriptomic Alterations Driven by Alphavirus-Based Cancer Immunotherapy Vectors

open access: yesJournal of Immunology Research
Cancer cells promote the polarisation of tumour-associated macrophages (TAMs) into pro-tumorigenic M2-like phenotype, contributing to cancer progression. Reprogramming TAMs by viral immunotherapy vectors represents a promising strategy for cancer therapy.
Ksenija Korotkaja   +3 more
doaj   +1 more source

Gene therapy for arthritis [PDF]

open access: yesExpert Opinion on Investigational Drugs, 1994
In the two years since arthritis gene therapy was last reviewed in this journal, there has been rapid progress on several fronts. Although vector development remains a slow process and long-term gene expression is not easily obtained, very encouraging preclinical data in animal models of arthritis are now emerging.
Paul D. Robbins, Christopher H. Evans
openaire   +4 more sources

B cell mechanobiology in health and disease: emerging techniques and insights into therapeutic responses

open access: yesFEBS Letters, EarlyView.
B cells sense external mechanical forces and convert them into biochemical signals through mechanotransduction. Understanding how malignant B cells respond to physical stimuli represents a groundbreaking area of research. This review examines the key mechano‐related molecules and pathways in B lymphocytes, highlights the most relevant techniques to ...
Marta Sampietro   +2 more
wiley   +1 more source

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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