摘要:
Genetically modified non-human animals and methods and compositions for making and using the same are provided, wherein the genetic modification comprises a humanization of an endogenous signal-regulatory protein gene, in particular a humanization of a SIRPα gene. Genetically modified mice are described, including mice that express a human or humanized SIRPα protein from an endogenous SIRPα locus.
摘要:
The invention provides a CRISPR/Cas9 composition for cleaving a target DNA in a plant cell, wherein the target DNA is endogenous DNA, and wherein the composition comprises (a) a nucleic acid encoding a CRISPR-associated protein 9 (Cas9) polypeptide, or a Cas9 polypeptide; and (b) a guide RNA specific to the target DNA, wherein the guide RNA is (i) a dual guide RNA comprising a CRISPR RNA (crRNA) and a trans activating crRNA (tracrRNA); or (ii) a single-chain guide RNA comprising a CRISPR RNA (crRNA) fused to a trans activating crRNA (tracrRNA). The invention further provides a plant cell, in particular a protoplast, and a plant, comprising the composition of the invention as well as a method for cleaving a target DNA or introducing two nicks into a target DNA of a plant cell using the composition of the invention.
摘要:
The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.
摘要:
The invention relates to an approach for introducing one or more desired insertions and/or deletions of known sizes into one or more predefined locations in a nucleic acid (eg, in a cell or organism genome). They developed techniques to do this either in a sequential fashion or by inserting a discrete DNA fragment of defined size into the genome precisely in a predefined location or carrying out a discrete deletion of a defined size at a precise location. The technique is based on the observation that DNA single-stranded breaks are preferentially repaired through the HDR pathway, and this reduces the chances of indels (eg, produced by NHEJ) in the present invention and thus is more efficient than prior art techniques. The invention also provides sequential insertion and/or deletions using single- or double-stranded DNA cutting.
摘要:
The use of P2E2 constructs in genome surgery includes a cell penetration component, a DNA binding component and a restriction endonuclease. The method for performing genome surgery includes: a) providing one or more recombinant of the P2E2 constructs; b) penetrating a cell with the recombinant P2E2 protein construct; c) forming a protein product in the cell by the processes of transcription and translation or by direct introduction of the P2E2 protein construct to the cell; d) attaching the protein product of the P2E2 construct to one or more targeted genomic sequences within the cell; and e) the endonuclease of the P2E2 construct cutting both strands of the genome at target locations.
摘要:
Nucleases and methods of using these nucleases for inserting a sequence encoding a therepeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.
摘要:
The present invention relates to stable and high-producing site- specific integration (SSI) host cells, e. g. Chinese hamster ovary (CHO)-derived host cells, methods to produce and to use them.
摘要:
The present invention provides; a novel gene introduction method which enables a gene to be introduced more safely and more freely, particularly a method for introducing a gene into a specified site in the brain safely and freely; a carrier for gene introduction use, which comprises a nano-particle and a substance capable of binding to a vector for gene introduction and has functional groups involved in the induction of phagocytosis by cells, wherein the substance capable of binding to a vector for gene introduction can bind to the surface of the nano-particle through some of the functional groups and another some of the functional groups remain unbound to the substance capable of binding to a vector for gene introduction ; and a gene introduction agent, in which a vector for gene introduction is bound to the substance capable of binding to a vector for gene introduction in the carrier for gene introduction.
摘要:
In some embodiments the present disclosure provides a composition for targeted alteration of a DNA sequence and methods of altering the targeted DNA sequence using the composition. In some embodiments such a composition comprises a MiniVector comprising a nucleic acid sequence template for homology-directed repair, alteration, or replacement of the targeted DNA sequence within a cell in vivo or in vitro, where the MiniVector lacks both a bacterial origin of replication and an antibiotic selection gene, and wherein the Mini Vector has a size up to about 2,500 base pairs.