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US 2023/0084201 A1Published application
SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF
AssigneeCedars-Sinai Medical Center
InventorsJoshua BREUNIG, Moise DANIELPOUR, Gi Bum KIM
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Abstract
Described herein are donor vectors and systems for use in in vivo dual recombinase-mediated cassette exchange. Also described are animal models for consistent, rigorous, and facile investigation of transgene expression. Further described are methods of screening for therapeutic drugs using these animal models, and methods of treatment.
Claims21
A system, comprising:
- a promoter-less donor vector, comprising
- a polyadenylation signal or transcription stop element upstream from a transgene or RNA,
- the transgene or RNA, and
- paired recombinase recognition sites;
- and
- one expression vector, comprising two genes encoding recombinases specific to the paired recombinase recognition sites, or
- two expression vectors, the first expression vector comprising one gene encoding a first recombinase that is specific to one of the paired recombinase recognition sites, and the second expression vector comprising one gene encoding a second recombinase that is specific to the other of the paired recombinase recognition sites.
4 more elements
A promoter-less donor vector, comprising:
- a polyadenylation signal or transcription stop element upstream from a transgene or RNA;
- the transgene or RNA;
- and
- paired recombinase recognition sites.
1 more element
A method of treating a disease or condition in a subject, comprising:
- providing a cell comprising a system, comprising:
- a promoter-less donor vector, comprising
- a polyadenylation signal or transcription stop element upstream from a transgene or RNA,
- the transgene or RNA, and
- paired recombinase recognition sites;
- and
- one expression vector, comprising two genes encoding recombinases specific to the paired recombinase recognition sites, or
- two expression vectors, the first expression vector comprising one gene encoding a first recombinase that is specific to one of the paired recombinase recognition sites, and the second expression vector comprising one gene encoding a second recombinase that is specific to the other of the paired recombinase recognition sites;
- and
- administering the cell to the subject.
7 more elements
(canceled)
Dependencies are inferred from the claim text.