Results 51 to 60 of about 1,902,725 (366)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

open access: yes, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

The immunological interface: dendritic cells as key regulators in metabolic dysfunction‐associated steatotic liver disease

open access: yesFEBS Letters, EarlyView.
Metabolic dysfunction‐associated steatotic liver disease (MASLD) affects nearly one‐third of the global population and poses a significant risk of progression to cirrhosis or liver cancer. Here, we discuss the roles of hepatic dendritic cell subtypes in MASLD, highlighting their distinct contributions to disease initiation and progression, and their ...
Camilla Klaimi   +3 more
wiley   +1 more source

Regulating Human Gene Therapy [PDF]

open access: yes, 1985
Scientific developments have moved the public debate on genetic engineering to the issue of human gene therapy. Because so many important societal values must be weighed in deciding which, if any, of the first protocols for human gene therapy should be ...
Areen, Judith C.
core   +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

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

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

Gene Therapy in Cardiac Arrhythmias [PDF]

open access: yes, 2006
Gene therapy has progressed from a dream to a bedside reality in quite a few human diseases. From its first application in adenosine deaminase deficiency, through the years, its application has evolved to vascular angiogenesis and cardiac arrhythmias.
Francis, Johnson   +2 more
core  

A PCA3 gene-based transcriptional amplification system targeting primary prostate cancer. [PDF]

open access: yes, 2015
Targeting specifically primary prostate cancer (PCa) cells for immune therapy, gene therapy or molecular imaging is of high importance. The PCA3 long non-coding RNA is a unique PCa biomarker and oncogene that has been widely studied.
Fradet, Yves   +5 more
core   +2 more sources

Inferring synthetic lethal interactions from mutual exclusivity of genetic events in cancer [PDF]

open access: yesBiology Direct 2015, 10:57, 2015
Background: Synthetic lethality (SL) refers to the genetic interaction between two or more genes where only their co-alteration (e.g. by mutations, amplifications or deletions) results in cell death. In recent years, SL has emerged as an attractive therapeutic strategy against cancer: by targeting the SL partners of altered genes in cancer cells, these
arxiv   +1 more source

Ablation of LRP6 in alpha‐smooth muscle actin‐expressing cells abrogates lung inflammation and fibrosis upon bleomycin‐induced lung injury

open access: yesFEBS Letters, EarlyView.
Low‐density lipoprotein receptor‐related protein 6 (LRP6) is a key receptor for the Wnt antagonist Dickkopf1 (DKK1). DKK1 protein expression is induced in a bleomycin (BLM)‐induced lung injury model. We show that DKK1 induces proinflammatory and profibrotic genes in lung fibroblasts.
Eun‐Ah Sung   +6 more
wiley   +1 more source

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