Abstract     Description     Claims     Drawing  

US8697359B   [0044] 
US8771945B   [0044] 
US8795965B   [0044] 
US8865406B   [0044] 
US8871445B   [0044] 
US8889356B   [0044] 
US8889418B   [0044] 
US8895308B   [0044] 
US20140310830A   [0044] 
US105031   [0044] 
US20140287938A1   [0044] 
US213991   [0044] 
US20140273234A1   [0044] 
US293674   [0044] 
US20140273232A1   [0044] 
US290575   [0044] 
US20140273231A   [0044] 
US259420   [0044] 
US20140256046A1   [0044] 
US226274   [0044] 
US20140248702A1   [0044] 
US258458   [0044] 
US20140242700A1   [0044] 
US222930   [0044] 
US20140242699A1   [0044] 
US183512   [0044] 
US20140242664A1   [0044] 
US104990   [0044] 
US20140234972A1   [0044] 
US183471   [0044] 
US20140227787A1   [0044] 
US256912   [0044] 
US20140189896A1   [0044] 
US105035   [0044] 
US20140186958A   [0044] 
US105017   [0044] 
US20140186919A1   [0044] 
US104977   [0044] 
US20140186843A1   [0044] 
US104900   [0044] 
US20140179770A1   [0044] 
US104837   [0044] 
US20140179006A1   [0044] 
US183486   [0044] 
US20140170753A   [0044] 
US183429   [0044] 
EP2771468A   [0044] 
EP13818570   [0044] 
EP2764103A   [0044] 
EP13824232   [0044] 
EP2784162A   [0044] 
EP14170383   [0044] 
WO2014093661A   [0044] 
US2013074743W   [0044] 
WO2014093694A   [0044] 
US2013074790W   [0044] 
WO2014093595A   [0044] 
US2013074611W   [0044] 
WO2014093718A   [0044] 
US2013074825W   [0044] 
WO2014093709A   [0044] 
US2013074812W   [0044] 
WO2014093622A   [0044]  [0091]  [0091]  [0095]  [0099]  [0102] 
US2013074667W   [0044]  [0091]  [0091]  [0095]  [0099]  [0102] 
WO2014093635A   [0044] 
US2013074691W   [0044] 
WO2014093655A   [0044] 
US2013074736W   [0044] 
WO2014093712A   [0044] 
US2013074819W   [0044] 
WO2014093701A   [0044] 
US2013074800W   [0044] 
WO2014018423A   [0044] 
US2013051418W   [0044] 
US2014041803W   [0044] 
US2014041800W   [0044] 
US2014041809W   [0044] 
US2014041804W   [0044] 
US2014041806W   [0044] 
US2014041808W   [0044] 
US201462558W   [0044] 
US1441806W   [0044]  [0044] 
US2014070057W   [0047] 
US8454972B2   [0116] 
US8404658B2   [0117] 
US5593972A   [0120]  [0256] 
US5589466A   [0120]  [0256] 
US5580859A   [0120]  [0256] 
US5049386A   [0125] 
US4946787A   [0125]  [0125] 
US4897355A   [0125] 
WO9117424A   [0125] 
WO9116024A   [0125] 
US4186183A   [0125] 
US4217344A   [0125] 
US4235871A   [0125] 
US4261975A   [0125] 
US4485054A   [0125] 
US4501728A   [0125] 
US4774085A   [0125] 
US4837028A   [0125] 
US9405700W   [0125] 
US20120164118A   [0125] 
US4797368A   [0125] 
WO9324641A   [0125] 
US5173414A   [0125] 
US20030087817A   [0126] 
US8454972B   [0132]  [0132] 
US8404658B   [0132]  [0132] 
US5846946A   [0132]  [0132] 
US20120295960A   [0142] 
US7303910B   [0142] 
US7351585B   [0142] 
US20060281180A   [0142] 
US20090007284A   [0142] 
US20110117189A   [0142] 
US20090017543A   [0142] 
US20070054961A   [0142] 
US20100317109A   [0142] 
US20110293571A   [0142]  [0142] 
US20040013648A   [0142] 
US20070025970A   [0142] 
US20090111106A   [0142] 
US7259015B   [0142] 
US8709843B   [0149]  [0173] 
US6007845A   [0149]  [0173] 
US5855913A   [0149]  [0173] 
US5985309A   [0149]  [0173] 
US5543158A   [0149]  [0173] 
US20110293703A   [0155]  [0156]  [0157] 
US20130302401A   [0158]  [0158] 
WO2012135025A   [0173] 
US20120251560A   [0173] 
US7982027B   [0195] 
US7799565B   [0195] 
US8058069B   [0195] 
US8283333B   [0195] 
US7901708B   [0195] 
US7745651B   [0195] 
US7803397B   [0195] 
US8101741B   [0195] 
US8188263B   [0195] 
US7915399B   [0195] 
US8236943B   [0195] 
US7838658B   [0195] 
EP1766035A   [0195] 
EP1519714A   [0195] 
EP1781593A   [0195] 
EP1664316A   [0195] 
US20130252281A   [0196] 
US20130245107A   [0196] 
US20130244279A   [0196] 
US20120251618A   [0196] 
US20050019923A   [0198]  [0199] 
US20080267903A   [0199] 
US8372951B   [0206] 
US8575305B   [0206] 
US8614194B   [0206] 
US8044019B   [0206] 
US20110195123A   [0207]  [0208]  [0208]  [0212]  [0213]  [0220] 
US20110230839A   [0227]  [0227] 
US20130171732A   [0232] 
US20100291048A   [0232] 
US20130137104A   [0232] 
US20130122591A   [0232] 
US20100146651A   [0232] 
WO2013126794A   [0239] 
US20110225664A   [0240]  [0240]  [0355] 
US20110091441A   [0240]  [0240] 
US20100229252A   [0240]  [0240] 
US20090271881A   [0240]  [0240] 
US20090222937A   [0240]  [0240] 
US20110182867A   [0244]  [0244] 
US20120328580A   [0247]  [0252] 
US20060030837A   [0248] 
US7206639B   [0248] 
US20070093878A   [0249] 
US20050287127A   [0253] 
US953797   [0253] 
US2007084654W   [0253] 
US20110142917A   [0255] 
US20120204282A   [0278] 
US20130183282A   [0279] 
US20130202678A   [0280] 
US20120159653A   [0282]  [0283] 
US20110023139A   [0311] 
WO9703211A   [0331] 
WO9639154A   [0331] 
US81573004   [0335] 
US20040171156A1   [0335] 
US491026   [0337] 
WO2011028929A   [0337] 
US511940   [0337] 
US4873316A   [0341] 
EP264166A   [0341] 
US6750059B   [0341] 
US092085   [0341] 
US7776321B   [0341] 
US20110059502A   [0342] 
US61736465B   [0343] 
US61721283B   [0343] 
US20110158957A   [0355] 
US20100311124A   [0355] 
US20110023145A   [0359]  [0360] 
US20110023153A   [0363] 
US20110023146A   [0369] 
US20110023144A   [0371] 

Correction of a Genetic Disease in a Mouse via Use of CRISPR-Cas9   [0004] 
Multiplex genome engineering using CRISPR/Cas systems   [0045] 
RNA-guided editing of bacterial genomes using CRISPR-Cas systems   [0045] 
One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering   [0045] 
Optical control of mammalian endogenous transcription and epigenetic states   [0045] 
Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity   [0045] 
DNA targeting specificity of RNA-guided Cas9 nucleases   [0045] 
Genome engineering using the CRISPR-Cas9 system   [0045] 
Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells   [0045] 
Crystal structure of cas9 in complex with guide RNA and target DNA   [0045] 
Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells   [0045] 
CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling   [0045] 
Development and Applications of CRISPR-Cas9 for Genome Engineering   [0045] 
Genetic screens in human cells using the CRISPR/Cas9 system   [0045] 
Rational design of highly active sgRNAs for CRISPR-Cas9-mediated gene inactivation   [0045] 
In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9   [0045] 
Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing   [0046] 
Nature   [0051] 
Small molecule therapy for genetic disease   [0051] 
Nature reviews. Genetics   [0052] 
Silence   [0052] 
Science translational medicine   [0052] 
The Journal of clinical investigation   [0052]  [0052] 
Science   [0052] 
Hematopoietic Stem Cell Expansion and Gene Therapy   [0053] 
Broadening the Indications for Hematopoietic Stem Cell Genetic Therapies   [0054] 
Hematopoietic stem cell-based gene therapy for HIV disease   [0055] 
Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9   [0055] 
CRISPR/Cas9 system to suppress HIV-1 expression by editing HIV-1 integrated proviral DNA   [0055] 
Nature reviews. Genetics   [0056] 
Science   [0056] 
Cell   [0056] 
Proceedings of the National Academy of Sciences of the United States of America   [0058]  [0058] 
Annual review of biophysics and biomolecular structure   [0058] 
Science   [0058]  [0060] 
Genetics   [0058]  [0059] 
The EMBO journal   [0058] 
Nature biotechnology   [0058] 
Science   [0058] 
Science   [0058] 
Genetics   [0058]  [0058] 
Nature biotechnology   [0058] 
Microbiology   [0058] 
Science   [0058] 
Nature   [0058] 
Nature   [0058] 
Nucleic acids research   [0058]  [0089] 
Science   [0058]  [0089] 
Proceedings of the National Academy of Sciences of the United States of America   [0058]  [0089] 
Science   [0058]  [0089] 
Science   [0058] 
Nature methods   [0058] 
Biotechnology and bioengineering   [0058] 
The New England journal of medicine   [0058]  [0067]  [0081] 
Nature   [0058]  [0067]  [0082] 
Nature   [0058]  [0065]  [0067]  [0070]  [0084]  [0085]  [0111] 
Nature biotechnology   [0058]  [0067]  [0070]  [0086] 
Cell   [0059] 
Molecular and cellular biology   [0060] 
Molecular and cellular biology   [0060] 
Nucleic acids research   [0060] 
Genetics   [0060] 
Molecular and cellular biology   [0060] 
Genetics   [0060] 
Immunological reviews   [0064] 
hematology   [0064]  [0065] 
Proceedings of the National Academy of Sciences of the United States of America   [0064] 
The New England journal of medicine   [0064]  [0070] 
Lancet   [0064]  [0070] 
Gene   [0064] 
Proceedings of the National Academy of Sciences of the United States of America   [0064] 
Molecular therapy: the journal of the American Society of Gene Therapy   [0064] 
Journal of internal medicine   [0065]  [0084] 
Nature biotechnology   [0067]  [0081] 
Nature biotechnology   [0067]  [0080] 
Proceedings of the National Academy of Sciences of the United States of America   [0067] 
Molecular therapy: the journal of the American Society of Gene Therapy   [0067] 
Science   [0067] 
Molecular therapy : the journal of the American Society of Gene Therapy   [0067] 
Nucleic acids   [0067] 
Cell stem cell   [0067] 
Cell stem cell   [0067] 
Molecular cell   [0069] 
Molecular cell   [0069] 
G3   [0070] 
Proceedings of the National Academy of Sciences of the United States of America   [0070] 
Pathophysiology of blood disorders   [0072] 
Nature reviews. Genetics   [0074]  [0086] 
Gene therapy   [0074] 
Molecular therapy: the journal of the American Society of Gene Therapy   [0074] 
Gene therapy   [0075] 
Review Article, Disrupted Signaling through the Fanconi Anemia Pathway Leads to Dysfunctional Hematopoietic Stem Cell Biology: Underlying Mechanisms and Potential Therapeutic Strategies   [0077] 
Cell   [0079] 
The New England journal of medicine   [0079] 
Molecular therapy : the journal of the American Society of Gene Therapy   [0081] 
Blood   [0082] 
Circulation research   [0086] 
Nature reviews. Genetics   [0086] 
Nature methods   [0088] 
Nature biotechnology   [0088] 
Nature methods   [0088]  [0110] 
Nature biotechnology   [0089] 
Genome research   [0089] 
Nature biotechnology   [0089] 
Nature biotechnology   [0089]  [0090] 
Nature biotechnology   [0089] 
Cell stem cell   [0089] 
Nature methods   [0089] 
Cell   [0090]  [0108] 
Nature biotechnology   [0090] 
Nature biotechnology   [0090] 
Nature biotechnology   [0090] 
Nucleic Acids Res.   [0091] 
Cell   [0091] 
Nature Biotechnology   [0091] 
The CRISPR system: small RNA-guided defence in bacteria and archaea   [0097] 
Codon Usage Database   [0099] 
Codon usage tabulated from the international DNA sequence databases: status for the year 2000   [0099] 
Nature biotechnology   [0108]  [0112] 
Nucleic acids research   [0108] 
Nature biotechnology   [0110]  [0112] 
Nature methods   [0110] 
Gene   [0111] 
Annual Review of Virology   [0111] 
Nature biotechnology   [0111] 
REMINGTON'S PHARMACEUTICAL SCIENCES   [0115] 
FEBS Let.   [0120]  [0257] 
Nat. Biotech.   [0120]  [0257] 
Mol. Vision.   [0120]  [0257] 
J. Mol. Biol.   [0120]  [0257] 
Nat. Gen.   [0120]  [0257] 
NAR   [0120]  [0257] 
Retina   [0120]  [0257] 
Lipid-like nanoparticles for small interfering RNA delivery to endothelial cells   [0122] 
Lipid-based nanotherapeutics for siRNA delivery   [0122] 
Exosome-mediated delivery of siRNA in vitro and in vivo   [0122] 
HUMAN GENE THERAPY   [0122] 
HUMAN GENE THERAPY   [0122] 
Science   [0125] 
TIBTECH   [0125] 
TIBTECH   [0125] 
TIBTECH   [0125] 
Nature   [0125] 
Biotechnology   [0125] 
Restorative Neurology and Neuroscience   [0125] 
British Medical Bulletin   [0125] 
Current Topics in Microbiology and Immunology   [0125] 
Gene Therapy   [0125] 
Science   [0125] 
Cancer Gene Ther.   [0125] 
Bioconjugate Chem.   [0125] 
Bioconjugate Chem.   [0125] 
Gene Therapy   [0125] 
Cancer Res.   [0125] 
J. Virol.   [0125] 
J. Virol.   [0125] 
Virol.   [0125] 
J. Virol.   [0125] 
J. Virol.   [0125] 
Am. J. Vet. Res.   [0125]  [0125] 
Am. J. Tropical Med. & Hygiene   [0125] 
Virology   [0125] 
Human Gene Therapy   [0125] 
Clin. Invest.   [0125] 
Mol. Cell. Biol.   [0125] 
Mol. Cell. Biol.   [0125] 
PNAS   [0125] 
J. Virol.   [0125] 
J. Virol.   [0125]  [0136] 
Genetics.   [0127] 
J Gene Med   [0140] 
HUMAN GENE THERAPY   [0140]  [0273] 
Sci Trans1 Med   [0141] 
Nature Nanotechnology   [0149]  [0174] 
In vitro and in vivo mRNA delivery using lipid-enveloped pH-responsive polymer nanoparticles   [0152] 
ACSNano   [0153] 
Mol Pharm   [0153] 
J Contr Rel   [0153] 
Mol Pharm   [0153] 
Mol Pharm   [0153] 
J Biophotonics   [0153] 
J Raman Spect   [0153] 
J Royal Soc Interface   [0153] 
Expert Opin Drug Deliv   [0153] 
Biomacromolecules   [0153] 
Int J Pharm   [0153] 
Proc Natl Acad Sci U S A.   [0154] 
Adv Mater.   [0154] 
Nano Lett.   [0154] 
ACS Nano.   [0154] 
ACS Nano.   [0154] 
Nat Nanotechnol.   [0154] 
N Engl J Med   [0159] 
Cancer Discovery   [0160] 
Molecular Therapy   [0161]  [0161]  [0162]  [0163] 
J. Am. Chem. Soc.   [0165] 
Small.   [0165] 
ACS Nano.   [0165] 
J. Am. Chem. Soc.   [0165] 
Nano Lett.   [0165] 
Proc. Natl. Acad. Sci. USA.   [0165] 
Nanomedicine   [0165] 
J. Am. Chem. Soc.   [0165] 
Nature   [0165] 
Proc. Natl. Acad. Sci. USA.   [0165] 
Sci. Transl. Med.   [0165] 
Small   [0165] 
Nucleic Acids Research   [0166] 
PNAS   [0167] 
Nature   [0168] 
Nat Biotechnol   [0175] 
Nature Protocols   [0180] 
Nucleic Acids Research   [0181]  [0238] 
Journal of Drug Delivery   [0183] 
Journal of Drug Delivery   [0183]  [0184]  [0184] 
Nature Biotechnology   [0186] 
Nature Letters   [0186]  [0186] 
Gene Therapy   [0187] 
Lancet   [0187] 
J. Clin. Invest.   [0187] 
Advanced Drug Delivery Reviews   [0188] 
Nature Niotechnology   [0191] 
Angew. Chem. Int. Ed.   [0192] 
Expression of therapeutic proteins after delivery of chemically modified mRNA in mice   [0193] 
Molecular Therapy-Nucleic Acids   [0194] 
Delivery and Formulation of Engineered Nucleic Acids   [0196] 
ACS Nano.   [0197] 
J Pharm Sci.   [0197] 
J Control Release   [0197] 
Journal of the American Chemical Society   [0200] 
Nat. Biotech.   [0201] 
Proc. Natl. Acad. Sci. USA   [0201]  [0203] 
ACS Chemical Biology   [0203] 
Chemistry & Biology   [0203] 
Methods in Enzymology   [0203] 
Chemistry & Biology   [0203] 
Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA   [0206] 
Clin Orthop Relat Res.   [0229] 
Rationally engineered therapeutic proteins with reduced immunogenicity   [0235] 
Lipid-like nanoparticles for small interfering RNA   [0238] 
Review Article, The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia   [0239] 
Outcomes of Gene Therapy for β-Thalassemia Major via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q-Globin Vector   [0239] 
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia   [0239] 
Development of Gene Therapy for Thalassemia   [0239] 
Seamless gene correction of β-thalassaemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyback   [0239] 
Annu. Rev. Med.   [0241]  [0242]  [0242] 
Immunol. Rev.   [0241]  [0242]  [0242]  [0243] 
Lancet   [0242] 
Cell   [0242] 
Nature   [0242] 
Nat. Genet.   [0243] 
N. Engl. J. Med.   [0243] 
Acad. Sci.   [0243] 
Proc. Natl. Acad. Sci. USA   [0243] 
Blood   [0243] 
Blood   [0243] 
Blood   [0243] 
Blood   [0243] 
Blood   [0243] 
Blood   [0243] 
Blood   [0243] 
Nat. Med.   [0243] 
Hum. Gene Ther.   [0243] 
Blood   [0243] 
Science   [0243] 
Nat. Med.   [0243] 
Lancet   [0243] 
Science   [0244] 
Science   [0244] 
Drug Discovery Today   [0247] 
CDER Data Standards Manual   [0251] 
Cell   [0253] 
Cell   [0253] 
Nature   [0253] 
Science   [0253] 
Nat. Biotechnol.   [0253] 
Cell   [0253] 
Gene Therapy   [0258] 
Hear Res.   [0259] 
Antioxidants & Redox Signaling   [0260] 
Molecular Therapy   [0261] 
Sci. Trans1 Med.   [0262] 
PLoS One   [0263] 
Genetic Diseases of the Eye   [0270] 
J Gene Med   [0272]  [0272] 
Human Gene Therapy   [0274]  [0274] 
Molecular Therapy   [0276] 
Sci Transl Med   [0277] 
Cell Stem Cell   [0281] 
Am. J. Hum. Genet.   [0302] 
PNAS   [0310] 
Nature   [0310] 
Biomaterials   [0310] 
Molecular Therapy   [0315]  [0315] 
Am J Respir Crit Care Med   [0316] 
Cell Stem Cell   [0321] 
Molecular Therapy   [0323] 
Molecular Therapy   [0324] 
Gene Therapy   [0325] 
Molecular Therapy   [0326] 
Molecular Therapy-Nucleic Acids   [0327] 
Molecular Therapy   [0328] 
PNAS   [0330] 
Overview of principles of hybridization and the strategy of nucleic acid probe assay   [0331] 
Nucleic Acids Research   [0331] 
NUCLEIC ACIDS RESEARCH   [0331] 
J. Mol. Biol.   [0331] 
J. MOL. BIOL.   [0331] 
Nuc. Acids Research   [0331] 
Short Protocols in Molecular Biology   [0331] 
J. Mol. Biol.   [0331] 
Short Protocols in Molecular Biology   [0331] 
FEMS Microbiol Lett.   [0331] 
FEMS Microbiol Lett.   [0331] 
Gene   [0331] 
Protein sequence alignments: a strategy for the hierarchical analysis of residue conservation   [0331] 
The classification of amino acid conservation   [0331] 
PNAS   [0332] 
Trends Biotechnol.   [0332] 
GENE EXPRESSION TECHNOLOGY: METHODS IN ENZYMOLOGY   [0337]  [0338] 
Cell   [0337] 
Mol. Cell. Biol.   [0337] 
Proc. Natl. Acad. Sci. USA.   [0337] 
Gene   [0339] 
Gene   [0339] 
GENE EXPRESSION TECHNOLOGY: METHODS IN ENZYMOLOGY   [0339] 
EMBO J.   [0339] 
Cell   [0339] 
Gene   [0339] 
Mol. Cell. Biol.   [0339] 
Virology   [0339] 
Nature   [0340] 
EMBO J.   [0340] 
MOLECULAR CLONING: A LABORATORY MANUAL   [0340] 
Genes Dev.   [0341] 
Adv. Immunol.   [0341] 
EMBO J.   [0341] 
Cell   [0341] 
Cell   [0341] 
Proc. Natl. Acad. Sci. USA   [0341] 
Science   [0341] 
Science   [0341] 
Genes Dev.   [0341] 
J. Bacteriol.   [0341] 
J. Bacteriol.   [0341] 
Mol. Microbiol.   [0341] 
Emerg. Infect. Dis.   [0341] 
Biochim. Biophys. Acta   [0341] 
Mol. Microbiol.   [0341] 
OMICS J. Integ. Biol.   [0341] 
Mol. Microbiol.   [0341] 
J. Bacteriol.   [0341] 
Mol. Microbiol.   [0341] 
MOLECULAR CLONING: A LABORATORY MANUAL   [0344] 
CURRENT PROTOCOLS IN MOLECULAR BIOLOGY   [0344] 
METHODS IN ENZYMOLOGY   [0344] 
PCR 2: A PRACTICAL APPROACH   [0344] 
ANTIBODIES, A LABORATORY MANUAL   [0344] 
ANIMAL CELL CULTURE   [0344] 
Genetic Instabilities and Neurological Diseases   [0353] 
RNA Biol.   [0353] 
Genetics of Epilepsy and Genetic Epilepsies   [0354] 
MINI-SYMPOSIUM: X-Linked Adrenoleukodystrophypa, Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X-Linked Adrenoleukodystrophy   [0357] 
Eur. Child. Adolesc. Psychiatry   [0363] 
PLoS Genetics   [0363] 
Arch. Neurol.   [0364] 
In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9   [0379] 
N Engl. J. Med.   [0431] 
Mol. Ther.   [0431] 
Lancet   [0431]