Compostions for the treatment or prevention of neurodegenerative disorders, in particular Parkinson's disease
The present invention relates to a pharmaceutical composition for the treatment or prevention of neurodegenerative disorders, in particular neuro-degenerative disorders which involve elevated levels of reactive oxygen species (ROS) in neuronal cells, comprising a delivery system which comprises a nu...
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Zusammenfassung: | The present invention relates to a pharmaceutical composition for the treatment or prevention of neurodegenerative disorders, in particular neuro-degenerative disorders which involve elevated levels of reactive oxygen species (ROS) in neuronal cells, comprising a delivery system which comprises a nucleic acid sequence coding for a telomerase, in particular human telomerase (hTERT), or the corresponding telomerase protein, as well as the delivery system for use in the treatment of such diseases.The neuro-degenerative disorder may be, e.g., selected from the group comprising Alzheimer's disease, Huntington's disease, Creutzfeldt-Jakob's disease, amyotrophic lateral sclerosis (ALS) and Parkinson's disease.The delivery system of said pharmaceutical composition may comprise a functional telomerase protein, in particular a hTERT protein, as well as a carrier, and/or a nucleic acid sequence coding for a telomerase, in particular human telomerase (hTERT), which may be DNA, mRNA or a derivative thereof, as well as a carrier or vector, in particular selected from the group comprising viral vectors and non-viral vectors.Non-viral vectors may be, e.g., selected from the group comprising nanoparticles, liposomes, polymers, (poly)peptides, DNA nanostructures, plasmids, and minicircle DNA. Viral vectors may be in particular selected from the group comprising retroviral vectors such as adenoviruses, lentiviruses, other viral vectors such as HSV, Sendai virus, poxviruses, papilloma viruses and parvoviruses, in particular variants of adeno-associated virus (AAV).In one specific embodiment, the delivery system comprises an expression vector construct including an expression cassette comprising a functional telomerase gene as well as any regulatory sequences required for its expression. |
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