Viral particles for treatment of synucleinopathies such as Parkinson's disease by gene therapy

• Main Authors: GONZ ¨ ¢ LEZ ASQUINORA, GERARDO, LINDEN REX MURRAY, LANZIGO PEREZ JOSE LUIS
• Format: Patent
• Language: Chinese; English
• Published: 22.07.2022
• Subjects: 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; SPIRITS; VINEGAR; WINE

The present disclosure relates to viral particles for use in the treatment of synucleinopathies, in particular accidental Parkinson's disease, by gene therapy. More specifically, the present disclosure relates to a viral particle comprising a nucleic acid construct comprising a transgene encoding a glucocerebrosidase for use in the treatment of synucleinopathies by gene therapy in a subject in need thereof. 本公开内容涉及用于通过基因疗法治疗突触核蛋白病变、特别是偶发性帕金森病的病毒颗粒。更具体而言，本公开内容涉及用于在有需要的受试者中通过基因疗法治疗突触核蛋白病变的病毒颗粒，所述病毒颗粒包含核酸构建体，其包括编码葡萄糖脑苷脂酶的转基因。