TREATMENT OF NEUROMUSCULAR DISEASES VIA GENE THERAPY THAT EXPRESSES KLOTHO PROTEIN

The present disclosure provides gene constructs containing a nucleic acid sequences encoding a mammalian s-KL, operatively linked to a muscle cell-specific promoter, for use in the treatment of motor impairment that may manifest, for example, in a neuromuscular disorder or disease, utilizing viral a...

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Bibliographische Detailangaben
Hauptverfasser:	Grabulosa, Mireia Herrando, Franquesa, Sergi Verdés, Rodriguez, Miguel Chillon, Acebes, Xavier Navarro, Merino, Assumpció Bosch
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present disclosure provides gene constructs containing a nucleic acid sequences encoding a mammalian s-KL, operatively linked to a muscle cell-specific promoter, for use in the treatment of motor impairment that may manifest, for example, in a neuromuscular disorder or disease, utilizing viral and non-viral vectors with muscle cell and motor neuron tropisms containing gene constructs containing a nucleic acid sequence encoding a mammalian s-KL, operatively linked to a promoter such as a muscle cell-specific promoter, is delivered in pharmaceutical compositions containing the expression vector, isolated cells containing the expression vector, and methods of treating motor impairment and motor neuron diseases.