Abstract  Description  Claims  Drawing  Search report  Cited references 

US62038358   [0001] 
US62180699   [0001] 
US8999641B   [0138]  [0446]  [0449] 
US8993233B   [0138]  [0446]  [0449] 
US8945839B   [0138]  [0446]  [0449] 
US8932814B   [0138]  [0446]  [0449] 
US8906616B   [0138]  [0446]  [0449] 
US8895308B   [0138]  [0396]  [0446]  [0449] 
US8889418B   [0138]  [0396]  [0446]  [0449] 
US8889356B   [0138]  [0446]  [0449] 
US8871445B   [0138]  [0446]  [0449] 
US8865406B   [0138]  [0446]  [0449] 
US8795965B   [0138]  [0446]  [0449] 
US8771945B   [0138]  [0446]  [0449] 
US8697359B   [0138]  [0446]  [0449] 
US20140310830A   [0138] 
US105031   [0138] 
US20140287938A1   [0138] 
US213991   [0138] 
US20140273234A1   [0138] 
US293674   [0138] 
US20140273232A1   [0138] 
US290575   [0138] 
US20140273231A   [0138] 
US259420   [0138] 
US20140256046A1   [0138] 
US226274   [0138] 
US20140248702A1   [0138] 
US258458   [0138] 
US20140242700A1   [0138] 
US222930   [0138] 
US20140242699A1   [0138] 
US183512   [0138] 
US20140242664A1   [0138] 
US104990   [0138] 
US20140234972A1   [0138] 
US183471   [0138] 
US20140227787A1   [0138] 
US256912   [0138] 
US20140189896A1   [0138] 
US105035   [0138] 
US20140186958A   [0138] 
US105017   [0138] 
US20140186919A1   [0138] 
US104977   [0138] 
US20140186843A1   [0138] 
US104900   [0138] 
US20140179770A1   [0138] 
US104837   [0138] 
US20140179006A1   [0138] 
US183486   [0138] 
US20140170753A   [0138] 
US183429   [0138] 
EP2784162B1   [0138] 
EP2771468B1   [0138] 
EP2771468A   [0138] 
EP13818570   [0138] 
EP2764103A   [0138] 
EP13824232   [0138] 
EP2784162A   [0138] 
EP14170383   [0138] 
WO2014093661A   [0138] 
US2013074743W   [0138] 
WO2014093694A   [0138] 
US2013074790W   [0138] 
WO2014093595A   [0138] 
US2013074611W   [0138] 
WO2014093718A   [0138] 
US2013074825W   [0138] 
WO2014093709A   [0138] 
US2013074812W   [0138] 
WO2014093622A   [0138] 
US2013074667W   [0138] 
WO2014093635A   [0138] 
US2013074691W   [0138] 
WO2014093655A   [0138] 
US2013074736W   [0138] 
WO2014093712A   [0138] 
US2013074819W   [0138] 
WO2014093701A   [0138] 
US2013074800W   [0138] 
WO2014018423A   [0138] 
US2013051418W   [0138] 
WO2014204723A   [0138] 
US2014041790W   [0138] 
WO2014204724A   [0138] 
US2014041800W   [0138]  [0138] 
WO2014204725A   [0138] 
US2014041803W   [0138]  [0138] 
WO2014204726A   [0138] 
US2014041804W   [0138]  [0138] 
WO2014204727A   [0138] 
US2014041806W   [0138]  [0138] 
WO2014204728A   [0138] 
US2014041808W   [0138]  [0138] 
WO2014204729A   [0138] 
US2014041809W   [0138]  [0138] 
US61758468   [0138] 
US61802174B   [0138] 
US61806375B   [0138] 
US61814263B   [0138] 
US61819803B   [0138] 
US61828130B   [0138] 
US61836123   [0138] 
US61835931   [0138] 
US61835936B   [0138]  [0138] 
US61836127B   [0138]  [0138] 
US61836A   [0138] 
US101A   [0138] 
US61836080B   [0138]  [0138] 
US61835973B   [0138]  [0138] 
US61862468   [0138] 
US61862355B   [0138] 
US61871301B   [0138] 
US61960777B   [0138] 
US61961980B   [0138] 
US201462558W   [0138] 
US61915150   [0138] 
US61915301B   [0138] 
US61915267B   [0138]  [0138]  [0138] 
US61915260B   [0138]  [0138]  [0138] 
US61757972B   [0138] 
US61768959B   [0138] 
US61836101B   [0138] 
US61835931B   [0138] 
US62010888B   [0138] 
US62010879B   [0138] 
US62010329B   [0138] 
US62010441B   [0138] 
US61939228B   [0138]  [0142] 
US61939242B   [0138] 
US61980012B   [0138]  [0138] 
US62038358B   [0138] 
US62054490B   [0138]  [0139] 
US62055484B   [0138]  [0139] 
US62055460B   [0138]  [0138]  [0139] 
US62055487B   [0138]  [0138]  [0139] 
WO62069243A   [0138] 
US62055484   [0138] 
US61980012   [0138] 
US61939242   [0138] 
US1441806W   [0138]  [0138] 
US61930214   [0138]  [0138] 
US61915251   [0138]  [0138] 
US62091455B   [0139] 
US62096708B   [0139] 
US62091462B   [0139] 
US62096324B   [0139] 
US62091456B   [0139] 
US62091461B   [0139] 
US62094903B   [0139] 
US62096761B   [0139] 
US62098059B   [0139] 
US62096656B   [0139] 
US62096697B   [0139] 
US62098158B   [0139] 
US62151052B   [0139] 
US62087537B   [0139] 
US62054651B   [0139] 
US62067886B   [0139] 
US62054675B   [0139] 
US62054528B   [0139] 
US62055454B   [0139] 
US62087475B   [0139] 
US62087546B   [0139] 
US62098285B   [0139] 
US1470068W   [0142] 
US61915267   [0142] 
US8454972B2   [0190] 
US8404658B2   [0191] 
US5593972A   [0194] 
US5589466A   [0194] 
US5580859A   [0194] 
US8454972B   [0203]  [0203] 
US8404658B   [0203]  [0203] 
US5846946A   [0203]  [0203] 
US20120295960A   [0214] 
US7303910B   [0214] 
US7351585B   [0214] 
US20060281180A   [0214] 
US20090007284A   [0214] 
US20110117189A   [0214] 
US20090017543A   [0214] 
US20070054961A   [0214] 
US20100317109A   [0214] 
US20110293571A   [0214]  [0214] 
US20040013648A   [0214] 
US20070025970A   [0214] 
US20090111106A   [0214] 
US7259015B   [0214] 
US20110293703A   [0224]  [0225]  [0226] 
US20130302401A   [0227]  [0227] 
US8709843B   [0242]  [0252] 
US6007845A   [0243]  [0252] 
US5855913A   [0244]  [0252] 
US5985309A   [0245]  [0252] 
US5543158A   [0246]  [0252] 
WO2012135025A   [0247] 
US20120251560A   [0247] 
US7982027B   [0278] 
US7799565B   [0278] 
US8058069B   [0278] 
US8283333B   [0278] 
US7901708B   [0278] 
US7745651B   [0278] 
US7803397B   [0278] 
US8101741B   [0278] 
US8188263B   [0278] 
US7915399B   [0278] 
US8236943B   [0278] 
US7838658B   [0278] 
EP1766035A   [0278] 
EP1519714A   [0278] 
EP1781593A   [0278] 
EP1664316A   [0278] 
US20130252281A   [0279] 
US20130245107A   [0279] 
US20130244279A   [0279] 
US20120251618A   [0279] 
US20050019923A   [0281]  [0282] 
US20080267903A   [0282] 
US20110195123A   [0287]  [0288]  [0288]  [0292]  [0293]  [0300] 
WO9703211A   [0340] 
WO9639154A   [0340] 
US81573004   [0365] 
US20040171156A1   [0365] 
US491026   [0367] 
WO2011028929A   [0367] 
US511940   [0367] 
US4873316A   [0374] 
EP264166A   [0374] 
US6750059B   [0374] 
US092085   [0374] 
US7776321B   [0374] 
US20110059502A   [0395] 
US20140186919A   [0396] 
US20140242700A   [0396] 
US20140273234A   [0396] 
US20140335620A   [0396] 
WO2014093635A1   [0396] 
WO2014093635A9   [0396] 
US61736465B   [0396] 
US61721283B   [0396] 
US5049386A   [0397] 
US4946787A   [0397]  [0397] 
US4897355A   [0397] 
WO9117424A   [0397] 
WO9116024A   [0397] 
US4186183A   [0397] 
US4217344A   [0397] 
US4235871A   [0397] 
US4261975A   [0397] 
US4485054A   [0397] 
US4501728A   [0397] 
US4774085A   [0397] 
US4837028A   [0397] 
US9405700W   [0397] 
US4797368A   [0397] 
WO9324641A   [0397] 
US5173414A   [0397] 
US20030087817A   [0397] 
US6603061B   [0400]  [0475] 
US7868149B   [0400]  [0475] 
US20090100536A   [0400]  [0475] 
US5210015A   [0428] 
US5445934A   [0430] 
US61736527   [0446]  [0449] 
US61748427B   [0446]  [0449] 

Multiplex genome engineering using CRISPR/Cas systems   [0141] 
RNA-guided editing of bacterial genomes using CRISPR-Cas systems   [0141] 
One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering   [0141] 
Optical control of mammalian endogenous transcription and epigenetic states   [0141] 
Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity   [0141] 
DNA targeting specificity of RNA-guided Cas9 nucleases   [0141] 
Genome engineering using the CRISPR-Cas9 system   [0141] 
Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells   [0141] 
Crystal structure of cas9 in complex with guide RNA and target DNA   [0141] 
Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells   [0141] 
CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling   [0141] 
Development and Applications of CRISPR-Cas9 for Genome Engineering   [0141] 
Genetic screens in human cells using the CRISPR/Cas9 system   [0141] 
Rational design of highly active sgRNAs for CRISPR-Cas9-mediated gene inactivation   [0141] 
In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9   [0141] 
Genome-scale transcription activation by an engineered CRISPR-Cas9 complex   [0141]  [0141] 
A split-Cas9 architecture for inducible genome editing and transcription modulation   [0141]  [0141] 
Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis   [0141]  [0141] 
In vivo genome editing using Staphylococcus aureus Cas9   [0141]  [0141] 
Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing   [0141] 
Nature   [0143] 
PLoS Comput Biol   [0143] 
Prot Sci   [0143] 
Genetics   [0186]  [0198] 
REMINGTON'S PHARMACEUTICAL SCIENCES   [0189]  [0495] 
FEBS Let.   [0194] 
Nat. Biotech.   [0194] 
Mol. Vision.   [0194] 
J. Mol. Biol.   [0194] 
Nat. Gen.   [0194] 
NAR   [0194] 
Retina   [0194] 
Lipid-like nanoparticles for small interfering RNA delivery to endothelial cells   [0196]  [0324] 
Lipid-based nanotherapeutics for siRNA delivery   [0196] 
Exosome-mediated delivery of siRNA in vitro and in vivo   [0196]  [0324] 
HUMAN GENE THERAPY   [0196] 
HUMAN GENE THERAPY   [0196] 
J. Virol.   [0208] 
J Gene Med   [0212] 
HUMAN GENE THERAPY   [0212] 
Sci Transl Med   [0213] 
In vitro and in vivo mRNA delivery using lipid-enveloped pH-responsive polymer nanoparticles   [0221] 
ACSNano   [0222] 
Mol Pharm   [0222] 
J Contr Rel   [0222] 
Mol Pharm   [0222] 
Mol Pharm   [0222] 
J Biophotonics   [0222] 
J Raman Spect   [0222] 
J Royal Soc Interface   [0222] 
Expert Opin Drug Deliv   [0222] 
Biomacromolecules   [0222] 
Int J Pharm   [0222] 
Proc Natl Acad Sci USA.   [0223] 
Adv Mater.   [0223] 
Nano Lett.   [0223] 
ACS Nano   [0223] 
ACS Nano   [0223] 
Nat Nanotechnol.   [0223] 
N Engl J Med   [0228] 
Cancer Discovery   [0229] 
Molecular Therapy   [0230]  [0230]  [0231]  [0232] 
J. Am. Chem. Soc.   [0234] 
Small   [0234] 
ACS Nano   [0234] 
J. Am. Chem. Soc.   [0234] 
Nano Lett.   [0234] 
Proc. Natl. Acad. Sci. USA.   [0234] 
Nanomedicine   [0234] 
J. Am. Chem. Soc.   [0234] 
Nature   [0234] 
Proc. Natl. Acad. Sci. USA.   [0234] 
Sci. Transl. Med.   [0234] 
Small   [0234] 
Nucleic Acids Research   [0235] 
PNAS   [0236] 
Nature   [0237] 
Nature Nanotechnology   [0248]  [0252] 
Nat Biotechnol   [0254] 
Nature Protocols   [0259] 
Nucleic Acids Research   [0260] 
Journal of Drug Delivery   [0262]  [0263]  [0264]  [0265] 
Nature Biotechnology   [0267] 
Nature Letters   [0267]  [0267] 
Gene Therapy   [0268] 
Lancet   [0269] 
J. Clin. Invest.   [0270] 
Advanced Drug Delivery Reviews   [0271] 
Nature Niotechnology   [0274] 
Angew. Chem. Int. Ed.   [0275] 
Expression of therapeutic proteins after delivery of chemically modified mRNA in mice   [0276] 
Molecular Therapy-Nucleic Acids   [0277] 
ACS Nano   [0280] 
J Pharm Sci.   [0280] 
J Control Release   [0280] 
Journal of the American Chemical Society   [0283] 
Nat. Biotech.   [0284] 
Proc. Natl. Acad. Sci. USA   [0284]  [0286] 
ACS Chemical Biology   [0286] 
Chemistry & Biology   [0286] 
Methods in Enzymology   [0286] 
Chemistry & Biology   [0286] 
Nature   [0307] 
Nature Biotech   [0318] 
Antibody-mediated targeting of siRNA via the human insulin receptor using avidin-biotin technology   [0323] 
Global non-viral gene transfer to the primate brain following intravenous administration   [0324] 
Lipid-based nanotherapeutics for siRNA delivery   [0324] 
Rationally engineered therapeutic proteins with reduced immunogenicity   [0330]  [0495] 
Overview of principles of hybridization and the strategy of nucleic acid probe assay   [0345] 
Nucleic Acids Research   [0352] 
NUCLEIC ACIDS RESEARCH   [0352] 
J. Mol. Biol.   [0352] 
J. MOL. BIOL.   [0352] 
Nuc. Acids Research   [0356] 
Short Protocols in Molecular Biology   [0356] 
J. Mol. Biol.   [0356] 
Short Protocols in Molecular Biology   [0356] 
FEMS Microbiol Lett.   [0356] 
FEMS Microbiol Lett.   [0356] 
Gene   [0358] 
Protein sequence alignments: a strategy for the hierarchical analysis of residue conservation   [0359] 
The classification of amino acid conservation   [0359] 
PNAS   [0361] 
Trends Biotechnol.   [0361] 
MOLECULAR CLONING: A LABORATORY MANUAL   [0362] 
CURRENT PROTOCOLS IN MOLECULAR BIOLOGY   [0362] 
A PRACTICAL APPROACH   [0362] 
ANTIBODIES, A LABORATORY MANUAL   [0362] 
ANIMAL CELL CULTURE   [0362] 
GENE EXPRESSION TECHNOLOGY: METHODS IN ENZYMOLOGY   [0367]  [0368] 
Cell   [0367] 
Mol. Cell. Biol.   [0367] 
Proc. Natl. Acad. Sci. USA.   [0367] 
Gene   [0369] 
Gene   [0370] 
GENE EXPRESSION TECHNOLOGY: METHODS IN ENZYMOLOGY   [0370] 
EMBO J.   [0371] 
Cell   [0371] 
Gene   [0371] 
Mol. Cell. Biol.   [0372] 
Virology   [0372] 
Nature   [0373] 
EMBO J.   [0373] 
MOLECULAR CLONING: A LABORATORY MANUAL   [0373] 
Genes Dev.   [0374] 
Adv. Immunol.   [0374] 
EMBO J.   [0374] 
Cell   [0374] 
Cell   [0374] 
Proc. Natl. Acad. Sci. USA   [0374] 
Science   [0374] 
Science   [0374] 
Genes Dev.   [0374] 
J. Bacteriol.   [0375] 
J. Bacteriol.   [0375] 
Mol. Microbiol.   [0375] 
Emerg. Infect. Dis.   [0375] 
Biochim. Biophys. Acta   [0375] 
Mol. Microbiol.   [0375] 
OMICS J. Integ. Biol.   [0375] 
Mol. Microbiol.   [0375] 
J. Bacteriol.   [0375] 
Mol. Microbiol.   [0375] 
The CRISPR system: small RNA-guided defence in bacteria and archaea   [0382] 
Codon Usage Database   [0385] 
Codon usage tabulated from the international DNA sequence databases: status for the year 2000   [0385] 
Nucleic Acids Res.   [0392] 
Cell   [0392] 
Nature Biotechnology   [0392] 
Science   [0397] 
TIBTECH   [0397] 
TIBTECH   [0397] 
TIBTECH   [0397] 
Nature   [0397] 
Biotechnology   [0397] 
Restorative Neurology and Neuroscience   [0397] 
British Medical Bulletin   [0397] 
Current Topics in Microbiology and Immunology   [0397] 
Gene Therapy   [0397] 
Science   [0397] 
Cancer Gene Ther.   [0397] 
Bioconjugate Chem.   [0397] 
Bioconjugate Chem.   [0397] 
Gene Therapy   [0397] 
Cancer Res.   [0397] 
J. Virol.   [0397] 
J. Virol.   [0397] 
Virol.   [0397] 
J. Virol.   [0397] 
J. Virol.   [0397] 
Virology   [0397] 
Human Gene Therapy   [0397] 
J. Clin. Invest.   [0397] 
Mol. Cell. Biol.   [0397] 
Mol. Cell. Biol.   [0397] 
PNAS   [0397] 
J. Virol.   [0397] 
Plant genome editing made easy: targeted mutagenesis in model and crop plants using the CRISPR/Cas system   [0400] 
Efficient gene editing in tomato in the first generation using the CRISPR/Cas9 system   [0400] 
Targeted genome modification of crop plants using a CRISPR-Cas system   [0400] 
Efficient genome editing in plants using a CRISPR/Cas system   [0400] 
RNA-guided genome editing in plants using a CRISPR-Cas system   [0400] 
Gene targeting using the Agrobacterium tumefaciens-mediated CRISPR-Cas system in rice   [0400] 
Exploiting SNPs for biallelic CRISPR mutations in the outcrossing woody perennial Populus reveals 4-coumarate: CoA ligase specificity and Redundancy   [0400] 
Targeted DNA degradation using a CRISPR device stably carried in the host genome   [0400] 
Crop genomics: advances and applications   [0400] 
Clinical Immunology   [0440] 
Genetic Instabilities and Neurological Diseases   [0450] 
RNA Biol.   [0450] 
Genetics of Epilepsy and Genetic Epilepsies   [0451] 
Genetic Diseases of the Eye   [0452] 
Genetic Disorders   [0453] 
Genetic Brain Disorders   [0453] 
Crop genomics:advances and applications   [0475] 
A TALEN genome-editing system for generating human stem cell-based disease models   [0495] 
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations   [0495] 
Efficient TALEN-mediated gene knockout in livestock   [0495] 
Knockout Rats via Embryo Microinjection of Zinc-Finger Nucleases   [0495] 
Targeted mutagenesis in the silkworm Bombyx mori using zinc finger nuclease mRNA injection   [0495] 
Non-transgenic genome modifications in a hemimetabolous insect using zinc-finger and TAL effector nucleases   [0495] 
Chimeric nucleases stimulate gene targeting in human cells   [0495] 
An improved zinc-finger nuclease architecture for highly specific genome editing   [0495] 
Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)   [0495] 
Targeted genome editing across species using ZFNs and TALENs   [0495] 
Targeting DNA double-strand breaks with TAL effector nucleases   [0495]  [0495] 
Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription   [0495] 
A TALE nuclease architecture for efficient genome editing   [0495] 
FLASH assembly of TALENs for high-throughput genome editing   [0495] 
Breaking the code of DNA binding specificity of TAL-type III effectors   [0495] 
A simple cipher governs DNA recognition by TAL effectors   [0495] 
A transcription activator-like effector toolbox for genome engineering   [0495] 
CRISPR-Cas system and its role in phage-bacteria interactions   [0495] 
CRISPR-Cas, the immune system of bacteria and archaea   [0495] 
Evolution and classification of the CRISPR-Cas systems   [0495] 
CRISPR-Cas systems in bacteria and archaea: versatile small RNAs for adaptive defense and regulation   [0495] 
Multiplex genome engineering using CRISPR-Cas systems   [0495] 
RNA-guided human genome engineering via Cas9   [0495] 
RNA-programmed genome editing in human cells   [0495] 
Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease   [0495] 
The CRISPR-Cas bacterial immune system cleaves bacteriophage and plasmid DNA   [0495] 
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity   [0495] 
Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria   [0495] 
Genome editing with engineered zinc finger nucleases   [0495] 
Dissecting neural function using targeted genome engineering technologies   [0495] 
Establishment of HIV-1 resistance in CD4(+) T cells by genome editing using zinc-finger nucleases   [0495] 
Multiplex Genome Engineering Using CRISPR-Cas Systems   [0495] 
High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases   [0495] 
In vivo genome editing using a high-efficiency TALEN system   [0495] 
Conservative homologous recombination preferentially repairs DNA double-strand breaks in the S phase of the cell cycle in human cells   [0495] 
CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III   [0495]  [0495] 
The Streptococcus thermophilus CRISPR-Cas system provides immunity in Escherichia coli   [0495] 
Efficient genome editing in zebrafish using a CRISPR-Cas system   [0495] 
One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR-Cas-Mediated Genome Engineering   [0495] 
Generation of gene-modified mice via Cas9/RNA-mediated gene targeting   [0495] 
Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression   [0495] 
Expanding small RNA interference   [0495] 
Insertion of DNA sequences into the human chromosomal beta-globin locus by homologous recombination   [0495] 
High frequency targeting of genes to specific sites in the mammalian genome   [0495] 
The length of homology required for gene targeting in embryonic stem cells   [0495] 
A protocol for constructing gene targeting vectors: generating knockout mice for the cadherin family and beyond   [0495] 
A rapid and general assay for monitoring endogenous gene modification   [0495] 
Translocation capture sequencing: a method for high throughput mapping of chromosomal rearrangements   [0495] 
Nucleotide sequences in Xenopus 5S DNA required for transcription termination   [0495] 
Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers   [0495] 
Overcoming transcription activator-like effector (TALE) DNA binding domain sensitivity to cytosine methylation   [0495] 
A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity   [0495] 
Targeted genome editing in pluripotent stem cells using zinc-finger nucleases   [0495] 
RNA-guided editing of bacterial genomes using CRISPR-Cas systems   [0495] 
Die kinetik der invertinwirkung   [0495] 
De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks   [0495] 
Probable inference, the law of succession, and statistical inference   [0495] 
Retinal gene therapy with a large MYO7A cDNA using adeno-assocaited virus   [0495] 
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial   [0495] 
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B   [0495] 
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors   [0495] 
Two alternative promoters direct neuron-specific expression of the rat microtubule-associated protein 1B gene   [0495] 
Definition of an efficient synthetic poly(A) site   [0495] 
Production and titering of recombinant adeno-associated viral vectors   [0495] 
Developments in neuronal cell culture   [0495] 
Genome editing with RNA-guided Cas9 nuclease in zebrafish embryos   [0495] 
Mammalian base excision repair: the forgotten archangel   [0495] 
Heritable genome editing in C. elegans via a CRISPR-Cas9 system   [0495] 
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells   [0495] 
Genome Engineering of Drosophila with the CRISPR RNA-Guided Cas9 Nuclease   [0495] 
DNA targeting specificity of RNA-guided Cas9 nucleases   [0495] 
CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering   [0495] 
Obligate ligation-gated recombination (ObLiGaRe): custom-designed nuclease-mediated targeted integration through nonhomologous end joining   [0495] 
High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity   [0495] 
CRISPR provides acquired resistance against viruses in prokaryotes   [0495] 
CRISPR-Cas systems: Prokaryotes upgrade to adaptive immunity   [0495] 
Xanthomonas AvrBs3 family-type III effectors: discovery and function   [0495] 
Classification and evolution of type II CRISPR-Cas systems   [0495] 
Multiplex genome engineering using CRISPR/Cas systems   [0495] 
CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III   [0495] 
Mammalian base excision repair: the forgotten archangel   [0495] 
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells   [0495] 
The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA   [0495] 
Molecular mechanisms of CRISPR-mediated microbial immunity   [0495] 
Development and Applications of CRISPR-Cas9 for Genome Engineering   [0495] 
DNA targeting specificity of RNA-guided Cas9 nucleases   [0495] 
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity   [0495] 
The discovery of zinc fingers and their applications in gene regulation and genome manipulation   [0495] 
Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease   [0495] 
CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering   [0495] 
Short motif sequences determine the targets of the prokaryotic CRISPR defence system   [0495] 
Crystal structure of cas9 in complex with guide RNA and target DNA   [0495] 
High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity   [0495] 
Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity   [0495] 
One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering   [0495] 
RNA-guided genetic silencing systems in bacteria and archaea   [0495] 
Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells   [0495] 
In vivo genome editing using Staphylococcus aureus Cas9   [0495]