摘要:
Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and to its application for genome engineering and gene therapy.
摘要:
This invention relates to a method of constructing a villin gene hybrid by inserting an I-Sce I restriction site next to or within a gene or cDNA encoding a villin protein. The insertion site of the I-Sce I restriction site is chosen as to provide a first downstream part and a second upstream part from the site, containing at least twelve nucleotides of the gene or cDNA encoding the villin protein. Furthermore, the insertion of the restriction permits a high frequency of homologous recombination events. The villin gene hybrid may be used to transfect eukaryotic cells, and particularly, embryonic stem cells.
摘要:
A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y (SEQ ID NO: 23 is residues 1-153 of pdb Ig9y) and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y (SEQ ID NO: 23 is residues 1-153 of pdb Ig9y) in a single polypeptide.
摘要翻译:包含具有登录号pdb 1g9y(SEQ ID NO:23的氨基酸序列为pdb Ig9y的残基1-153)的I-CreI的第一变体的单链归巢内切核酸酶以及I-CreI变体的第二变体,其具有 在单个多肽中,登录号pdb 1g9y(SEQ ID NO:23是pdb Ig9y的残基1-153)的氨基酸序列。
摘要:
This patent application relates to hybrid and/or single-chain rare-cutting endonucleases, called meganucleases, which recognize and cleave a specific nucleotide sequence, to polynucleotide sequences encoding for said rare-cutting endonucleases, to a vector comprising one of said polynucleotide sequences, to a cell or animal comprising one of said polynucleotide sequences or said rare-cutting endonucleases, to a process for producing one of said rare-cutting endonucleases and any use of the disclosed products and methods. More particularly, this invention contemplates any use of such rare-cutting endonuclease for genetic engineering and gene therapy.
摘要:
Methods of modifying, repairing, attenuating and inactivating a gene or other chromosomal DNA in a cell are disclosed. Also disclosed are methods of treating or prophylaxis of a genetic disease in an individual in need thereof.
摘要:
Methods of modifying, repairing, attenuating and inactivating a gene or other chromosomal DNA in a cell are disclosed. Also disclosed are methods of treating or prophylaxis of a genetic disease in an individual in need thereof. Further disclosed are chimeric restriction endonucleases.
摘要:
The invention relates to a set of genetic constructs which allow the efficient and reproducible introduction of a specific nucleotide sequence at a fixed position in the genome by generating a double strand break at a specific position in the genome using a meganuclease and so stimulating a homologous recombination event at this locus between the genomic site and a transfected donor sequence. The present invention also relates to methods using these constructs and to these materials in the form of a kit.
摘要:
This patent application relates to hybrid and/or single-chain rare-cutting endonucleases, called meganucleases, which recognize and cleave a specific nucleotide sequence, to polynucleotide sequences encoding for said rare-cutting endonucleases, to a vector comprising one of said polynucleotide sequences, to a cell or animal comprising one of said polynucleotide sequences or said rare-cutting endonucleases, to a process for producing one of said rare-cutting endonucleases and any use of the disclosed products and methods. More particularly, this invention contemplates any use of such rare-cutting endonuclease for genetic engineering and gene therapy.
摘要:
A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y in a single polypeptide.
摘要:
A retroviral vector that eliminates cis-acting retroviral elements, which are not useful to and may cause problems for, an integrated provirus utilizes a recombination system, such as the bacteriophage P1 cre-lox recombination system. A gene of interest and a loxP site are inserted into a long terminal repeat, which is then duplicated. The enzyme cre is inserted between the long terminal repeats and combines them. The structure of the resulting provirus in the host genome is that of a single, long terminal repeat carrying a single copy of the gene of interest. Following infection, the retrovirus produces proviral structures having only a single long terminal repeat.