Results 31 to 40 of about 2,093,614 (359)

LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]

open access: yes, 2010
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA   +5 more
core   +3 more sources

Urocortin 2 Gene Transfer Improves Glycemic Control and Reduces Retinopathy and Mortality in Murine Insulin Deficiency. [PDF]

open access: yes, 2020
Type 1 diabetes affects 20 million patients worldwide. Insulin is the primary and commonly the sole therapy for type 1 diabetes. However, only a minority of patients attain the targeted glucose control and reduced adverse events.
Bhargava, Raag   +10 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

Gene therapy. [PDF]

open access: yesGut, 1982
Gene therapy is not yet possible, but may become feasible soon, particularly for well understood gene defects. Although treatment of a patient raises no ethical problems once it can be done well, changing the genes of an early embryo is more difficult, controversial and unlikely to be required clinically.
openaire   +4 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

Assembly and functional analysis of an S/MAR based episome with the cystic fibrosis transmembrane conductance regulator gene [PDF]

open access: yes, 2018
Improving the efficacy of gene therapy vectors is still an important goal toward the development of safe and efficient gene therapy treatments. S/MAR (scaffold/matrix attached region)-based vectors are maintained extra-chromosomally in numerous cell ...
Ascenzioni, Fiorentina   +10 more
core   +3 more sources

Review of precision cancer medicine: Evolution of the treatment paradigm. [PDF]

open access: yes, 2020
In recent years, biotechnological breakthroughs have led to identification of complex and unique biologic features associated with carcinogenesis. Tumor and cell-free DNA profiling, immune markers, and proteomic and RNA analyses are used to identify ...
Fountzilas, Elena   +3 more
core   +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

Surface immobilization of hexa-histidine-tagged adeno-associated viral vectors for localized gene delivery. [PDF]

open access: yes, 2010
Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical trials, have significant promise for therapeutic gene delivery because of their safety and delivery efficiency.
Bethi, SR   +5 more
core   +2 more sources

Gene therapy [PDF]

open access: yesOral Diseases, 2013
Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients ...
openaire   +2 more sources

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