Gene therapy progress and prospects: transcription regulatory systems

C Toniatti, H Bujard, R Cortese, G Ciliberto - Gene therapy, 2004 - nature.com
The clinical efficacy and safety as well as the application range of gene therapy will be
broadened by developing systems capable of finely modulating the expression of …

Nerve growth factor: from animal models of cholinergic neuronal degeneration to gene therapy in Alzheimer's disease

MH Tuszynski, A Blesch - Progress in Brain research, 2004 - Elsevier
Over the last 20 years it has been recognized that neurotrophic factors profoundly influence
the development of the nervous system and have the potential to modify disease processes …

Tetracycline‐aptamer‐mediated translational regulation in yeast

S Hanson, K Berthelot, B Fink… - Molecular …, 2003 - Wiley Online Library
We describe post‐transcriptional gene regulation in yeast based on direct RNA–ligand
interaction. Tetracycline‐dependent translational regulation could be imposed via specific …

Autoimmune anemia in macaques following erythropoietin gene therapy

P Chenuaud, T Larcher, JE Rabinowitz, N Provost… - Blood, 2004 - ashpublications.org
We delivered the homologous erythropoietin (Epo) cDNA driven from a doxycycline-
regulated promoter via recombinant adeno-associated virus in skeletal muscle of 9 …

Timed GDNF gene therapy using an immune-evasive gene switch promotes long distance axon regeneration

R Eggers, F De Winter, SA Hoyng, RC Hoeben… - Brain, 2019 - academic.oup.com
Neurosurgical repair in patients with proximal nerve lesions results in unsatisfactory
recovery of function. Gene therapy for neurotrophic factors is a powerful strategy to promote …

Gas-inducible transgene expression in mammalian cells and mice

W Weber, M Rimann, M Spielmann, B Keller… - Nature …, 2004 - nature.com
We describe the design and detailed characterization of a gas-inducible transgene control
system functional in different mammalian cells, mice and prototype biopharmaceutical …

[HTML][HTML] GDNF gene therapy to repair the injured peripheral nerve

R Eggers, F De Winter, MR Tannemaat… - … in bioengineering and …, 2020 - frontiersin.org
A spinal root avulsion is the most severe proximal peripheral nerve lesion possible. Avulsion
of ventral root filaments disconnects spinal motoneurons from their target muscles, resulting …

Developing strategies for detection of gene doping

A Baoutina, IE Alexander, JEJ Rasko… - The Journal of Gene …, 2008 - Wiley Online Library
It is feared that the use of gene transfer technology to enhance athletic performance, the
practice that has received the term 'gene doping', may soon become a real threat to the …

Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives

F Rolling - Gene therapy, 2004 - nature.com
Retinal degenerative diseases such as retinal macular degeneration and retinitis
pigmentosa constitute a broad group of diseases that all share one critical feature, the …

Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector

I Barde, MA Zanta-Boussif, S Paisant, M Leboeuf… - Molecular Therapy, 2006 - cell.com
This work addresses the problem of efficient control of gene expression in the context of viral
vectors, which still represents a difficult challenge. A number of lentiviral vectors …