The invention relates to the field of transposases In particular, it provides a polypeptide comprising or consisting of a Tc1/mariner superfamily transposase, e.g., a Sleeping Beauty transposase, which comprises a nuclear export signal and a chaperone-mediated autophagy signal. The transposases of the invention were found to exhibit reduced half-life and thus enhanced biosafety. The invention further provides a sequence-optimized RNA comprising a 5'UTR and a 3'UTR that encodes a transposase with high transposition efficiency. The transposase encoded by the RNA may be, e.g., a transposase of the invention that is characterized by a reduced half-life while retaining full integration efficiency. The invention also provides vectors and cells for the expression of said transposase and/or RNA, kits and pharmaceutical compositions comprising said transposase and/or RNA, e.g., for use in gene therapy, e.g., adoptive T cell therapy, or uses and methods for gene delivery into the genome of a cell.
- 출원번호 : EP2024/072369
- 출원인 : MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN IN DER HELMHOLTZ-GEMEINSCHAFT;
- 특허번호 :
- IPC : C12N-009/12(2006.01);C12N-015/63(2006.01);C12N-015/85(2006.01);C12N-015/90(2006.01);C12N-009/22(2006.01);C12N-015/67(2006.01);