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公开(公告)号：US20230323391A1
公开(公告)日：2023-10-12
申请号：US18003021
申请日：2021-06-29
发明人： Stuart Robert COBB , Paul Ross , Ralph David HECTOR , Susan ROSSER , Adam MOL
CPC分类号： C12N15/86 , C12N15/111 , C12N2750/14143 , C12N2330/51 , C12N2310/141 , C12N2320/53 , C12N2830/42 , C12N2830/50 , C12N2830/48
摘要： A system to limit the expression of a vector-derived transgene within a window that alleviates the disease-causing genetic deficiency without producing overexpression toxicity is described. This provides for ‘dosage-insensitivity’, whereby cells or tissues receiving more vector-derived transgene are disproportionately suppressed through an in-built single gene circuit that can regulate adaptively.
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公开(公告)号：US20210101938A1
公开(公告)日：2021-04-08
申请号：US16497271
申请日：2018-03-23
申请人： THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH , THE UNIVERSITY COURT OF THE UNIVERSITY OF GLASGOW
摘要： MeCP2 based therapy. The present invention relates to synthetic polypeptides that are useful in the treatment of disorders associated with reduced MeCP2 activity, including Rett syndrome. The present invention provides synthetic polypeptides comprising: i) an MBD amino acid sequence showing at least 70% similarity with the amino acid sequence showing at least 70% similarity with the amino acid sequence as depicted in SEQ ID NO: 2, wherein the polypeptide has a deletion of at least 50 amino acids, when compared to the full length MeCP2 e1 and e2 sequences. The invention further provides nucleic acid constructs, expression vectors, virions, pharmaceutical compositions, and cells providing polynucleotides of the invention. The invention further provides methods of treating or preventing disease in an animal comprising administering to said animal a synthetic polypeptide according to the invention.
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公开(公告)号：US20240165270A1
公开(公告)日：2024-05-23
申请号：US18503439
申请日：2023-11-07
申请人： THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH , THE UNIVERSITY COURT OF THE UNIVERSITY OF GLASGOW
CPC分类号： A61K48/0066 , A61K9/0019 , A61K9/0085 , A61P25/14 , C07K14/001 , C07K14/4703 , C12N7/00 , C12N15/86 , A61K38/00 , C12N2750/14143
摘要： MECP2 EXPRESSION CASSETTES. The present invention provides nucleic acid molecules comprising a MeCP2 expression cassette, the expression cassette comprising, in operable linkage from 5′ to 3′: a 5′ transcriptional control region comprising a promoter capable of driving transcription in neural cells; an open reading frame encoding a MeCP2 protein; translation control signals; a 3′ untranslated region (3′UTR) comprising one or more of: (i) a binding site for mir-22; (ii) a binding site for mir-19; (iii) a binding site for miR-132; (iv) a binding site for miR124; and (v) an AU-rich element; and transcriptional termination signals; wherein the MeCP2 expression cassette is not more than about 5 kb in length. The invention further provides viral vectors, especially vectors derived from adeno-associated virus (AAV), for use in therapeutic delivery of such expression cassettes. The nucleic acid molecules and viral vectors disclosed herein provide novel tools for expressing MeCP2 and are of particular value in the treatment of disorders associated with reduced MeCP2 activity, including Rett syndrome.
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公开(公告)号：US20200016279A1
公开(公告)日：2020-01-16
申请号：US16497277
申请日：2018-03-23
申请人： THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH , THE UNIVERSITY COURT OF THE UNIVERSITY OF GLASGOW
摘要： The present invention provides nucleic acid molecules comprising a MeCP2 expression cassette, the expression cassette comprising, in operable linkage from 5′ to 3′: a 5′ transcriptional control region comprising a promoter capable of driving transcription in neural cells; an open reading frame encoding a MeCP2 protein; translation control signals; a 3′ untranslated region (3′UTR) comprising one or more of: (i) a binding site for mir-22; (ii) a binding site for mir-19; (iii) a binding site for miR-132; (iv) a binding site for miR124; and (v) an AU-rich element; and transcriptional termination signals; wherein the MeCP2 expression cassette is not more than about 5 kb in length. The invention further provides viral vectors, especially vectors derived from adeno-associated virus (AAV), for use in therapeutic delivery of such expression cassettes. The nucleic acid molecules and viral vectors disclosed herein provide novel tools for expressing MeCP2 and are of particular value in the treatment of disorders associated with reduced MeCP2 activity, including Rett syndrome.
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