Patent
Methods and compositions for modifying genomic dna
Linhong Li,Madhusudan Peshwa +1 more
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TLDR
In this paper, the authors proposed a method for site-specific sequence modification of a target genomic DNA region in cells comprising: transfecting the cells by electroporation with a composition comprising a DNA oligo and a DNA digesting agent wherein the donor DNA comprises: (i) a homologous region comprising nucleic acid sequence homologously to the target DNA region and (ii) a sequence modification region.Abstract:
Compositions and methods concern the sequence modification of an endogenous genomic DNA region. Certain aspects relate to a method for site-specific sequence modification of a target genomic DNA region in cells comprising: transfecting the cells by electroporation with a composition comprising (a) a DNA oligo and (b) a DNA digesting agent wherein the donor DNA comprises: (i) a homologous region comprising nucleic acid sequence homologous to the target genomic DNA region and (ii) a sequence modification region; and wherein the genomic DNA sequence is modified specifically at the target genomic DNA region.read more
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Patent
CRISPR-related methods and compositions with governing gRNAs
Alexandra Glucksmann,Deborah Palestrant,Louis Anthony Tartaglia,Jordi Mata-Fink,Agnieszka Czechowicz,Borisy Alexis +5 more
TL;DR: In this article, methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas 9 molecule/gRNA complex.
Patent
Cas variants for gene editing
David R. Liu,Alexis C. Komor +1 more
TL;DR: In this paper, the authors present strategies, systems, reagents, methods and kits for targeted nucleic acid editing, including editing a single site within the genome of a cell or subject, eg, within the human genome.
Patent
Delivery system for functional nucleases
TL;DR: In this paper, the authors present methods, methods, strategies, kits, and systems for the supercharged protein- mediated delivery of functional effector proteins into cells in vivo, ex vivo, or in vitro.
Patent
Adenosine nucleobase editors and uses thereof
David R. Liu,Nicole M. Gaudelli +1 more
TL;DR: In this article, the authors provide fusion proteins comprising a Cas9 (e.g., a Cas 9 nickase) domain and adenosine deaminases that deaminate adenosines in DNA, and in some embodiments, the fusion proteins further comprise a nuclear localization sequence (NLS), and/or an inhibitor of base repair, such as, a nuclease dead inosine specific nucolate (dISN).
References
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Journal ArticleDOI
Genome engineering using the CRISPR-Cas9 system
F. Ann Ran,Patrick D. Hsu,Jason Wright,Vineeta Agarwala,Vineeta Agarwala,David A. Scott,Feng Zhang +6 more
TL;DR: A set of tools for Cas9-mediated genome editing via nonhomologous end joining (NHEJ) or homology-directed repair (HDR) in mammalian cells, as well as generation of modified cell lines for downstream functional studies are described.
Book
Molecular Cell Biology
TL;DR: Molecular cell biology, Molecular cell biology , مرکز فناوری اطلاعات و اصاع رسانی, کδاوρزی
Journal ArticleDOI
Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
TL;DR: Delivery of purified recombinant Cas9 protein and guide RNA into cultured human cells including hard-to-transfect fibroblasts and pluripotent stem cells is delivered and RGEN ribonucleoproteins (RNPs) induce site-specific mutations at frequencies of up to 79%, while reducing off- target mutations associated with plasmid transfection at off-target sites.
Patent
Uncharged morpholino-based polymers having achiral intersubunit linkages
TL;DR: In this article, a polymer composition is disclosed composed of morpholino subunit structures which are linked together by uncharged, achiral linkages, which are one to three atoms in length, joining the morpholine nitrogen of one subunit to the 5' exocyclic carbon of an adjacent subunit.
Book
Antisense Research and Applications
Sudhir Agrawal,Stanley T. Crooke +1 more
TL;DR: Pharmacological Effects of Antisense Oligonucleotides to Protein Kinase C-a and C-raf Kinase: Rationale and Clinical Experience in Patients with Solid Tumors and Nucleic Acid Therapeutics for Human Leukemia.