FIELD: biotechnology.
SUBSTANCE: disclosed is a composition containing a CRISPR-Cas system for use in treating a postmitotic cell disorder, where Cas9 contains at least one nuclear localization sequence. What is also presented is using the composition containing polynucleotides expressing Cas9, containing at least one nuclear localization sequence, a guide sequence, a paired tracr-sequence and a tracr-sequence, to obtain a composition, containing CRISPR-Cas system for use in treating postmitotic cell disorders.
EFFECT: this group of inventions provides targeted gene editing, in connection with which can be used in gene therapy.
30 cl, 76 dwg, 17 tbl, 38 ex
| Title | Year | Author | Number |
|---|---|---|---|
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| DELIVERY, CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND USE IN THERAPY | 2013 |
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| SYSTEM DESIGN, METHODS AND OPTIMIZED GUIDE COMPOSITIONS FOR SEQUENCE MANIPULATION | 2013 |
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| COMPOSITIONS AND METHODS OF TREATING HEMOGLOBINOPATHIES | 2016 |
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