AR125416A1 - AAVRH74 VECTORS FOR GENE THERAPY OF MUSCULAR DYSOTROPHIES - Google Patents
AAVRH74 VECTORS FOR GENE THERAPY OF MUSCULAR DYSOTROPHIESInfo
- Publication number
- AR125416A1 AR125416A1 ARP220101055A ARP220101055A AR125416A1 AR 125416 A1 AR125416 A1 AR 125416A1 AR P220101055 A ARP220101055 A AR P220101055A AR P220101055 A ARP220101055 A AR P220101055A AR 125416 A1 AR125416 A1 AR 125416A1
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- AR
- Argentina
- Prior art keywords
- sequence
- nucleic acid
- itr
- capsid protein
- acid vector
- Prior art date
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
Se proporcionan proteínas de la cápside de AAV modificadas, partículas, vectores de ácidos nucleicos y composiciones de las mismas, así como métodos para su uso. Reivindicación 1: Una proteína de la cápside que comprende una sustitución de aminoácido en una posición que corresponde a Y447, T494, K547, N665, y/o Y733 de la proteína de la cápside AAVrh74 de tipo salvaje de la SEQ ID Nº 1, en donde la proteína de la cápside es una proteína de la cápside serotipo AAVrh74, opcionalmente en donde la sustitución es Y447F, T494V, K547R, N665R, y/o Y733F. Reivindicación 3: La partícula de AAVrh74 de la reivindicación 2, que comprende además un vector de ácido nucleico, en donde el vector de ácido nucleico comprende una primera repetición terminal invertida (ITR) que comprende una primera secuencia D y una segunda ITR que comprende una segunda secuencia D, en donde la primera secuencia D o la segunda secuencia D se sustituye con una secuencia S, opcionalmente en donde la secuencia S comprende, consiste esencialmente en, o consiste en la secuencia de nucleótidos TATTAGATCTGATGGCCGCT (SEQ ID Nº 17). Reivindicación 4: La partícula de AAVrh74 de la reivindicación 2, que comprende además un vector de ácido nucleico, en donde el vector de ácido nucleico comprende una primera repetición terminal invertida (ITR) que comprende una primera secuencia D y una segunda ITR que comprende una segunda secuencia D, en donde la primera secuencia D y/o la segunda secuencia D se sustituye con un elemento de unión a receptor glucocorticoide (GRE), opcionalmente en donde el GRE comprende, consiste esencialmente en, o consiste en la secuencia de nucleótidos AGAACANNNTGTTCT (SEQ ID Nº 18), o su inverso o complemento inverso, en donde cada N es independientemente un T, C, G, o A.Modified AAV capsid proteins, particles, nucleic acid vectors, and compositions thereof, as well as methods for their use, are provided. Claim 1: A capsid protein comprising an amino acid substitution at a position corresponding to Y447, T494, K547, N665, and/or Y733 of the wild-type AAVrh74 capsid protein of SEQ ID NO:1, in where the capsid protein is a serotype AAVrh74 capsid protein, optionally where the substitution is Y447F, T494V, K547R, N665R, and/or Y733F. Claim 3: The rh74AAV particle of claim 2, further comprising a nucleic acid vector, wherein the nucleic acid vector comprises a first inverted terminal repeat (ITR) comprising a first D sequence and a second ITR comprising a second D sequence, wherein either the first D sequence or the second D sequence is substituted with an S sequence, optionally wherein the S sequence comprises, consists essentially of, or consists of the nucleotide sequence TATTAGATCTGATGGCCGCT (SEQ ID NO: 17). Claim 4: The rh74AAV particle of claim 2, further comprising a nucleic acid vector, wherein the nucleic acid vector comprises a first inverted terminal repeat (ITR) comprising a first D-sequence and a second ITR comprising a second D-sequence, wherein the first D-sequence and/or the second D-sequence is substituted with a glucocorticoid receptor (GRE) binding element, optionally wherein the GRE comprises, consists essentially of, or consists of the nucleotide sequence AGAACANNNTGTTCT (SEQ ID Nº 18), or its inverse or inverse complement, where each N is independently a T, C, G, or A.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063327410P | 2020-04-05 | 2020-04-05 | |
US202163179097P | 2021-04-23 | 2021-04-23 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR125416A1 true AR125416A1 (en) | 2023-07-12 |
Family
ID=87551293
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP220101055A AR125416A1 (en) | 2020-04-05 | 2022-04-22 | AAVRH74 VECTORS FOR GENE THERAPY OF MUSCULAR DYSOTROPHIES |
Country Status (1)
Country | Link |
---|---|
AR (1) | AR125416A1 (en) |
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2022
- 2022-04-22 AR ARP220101055A patent/AR125416A1/en unknown
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