Targeted CRISPR delivery platforms
The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hvperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide...
Gespeichert in:
| Hauptverfasser: | , , , , , |
|---|---|
| Format: | Patent |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext bestellen |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | |
|---|---|
| container_issue | |
| container_start_page | |
| container_title | |
| container_volume | |
| creator | ILDAR, Gainetdinov IBRAHEIM, Raed SONTHEIMER, Erik Joseph EDRAKI, Alireza XUE, Wen MIR, Aamir |
| description | The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hvperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide RNA. sequences that permit a complete sgRNA-Nme1Cas9 vector to be inserted into an adeno-associated viral plasmid that is compatible for in vivo administration. Furthermore, Type II-C Cas9 oithologs have been identified that target protospacer adjacent motif sequences limited to between one - four required nucleotides. |
| format | Patent |
| fullrecord | <record><control><sourceid>epo_EVB</sourceid><recordid>TN_cdi_epo_espacenet_AU2018364993A1</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>AU2018364993A1</sourcerecordid><originalsourceid>FETCH-epo_espacenet_AU2018364993A13</originalsourceid><addsrcrecordid>eNrjZFAKSSxKTy1JTVFwDvIMDghSSEnNySxLLapUKMhJLEnLL8ot5mFgTUvMKU7lhdLcDMpuriHOHrqpBfnxqcUFicmpeakl8Y6hRgaGFsZmJpaWxo6GxsSpAgAZsiaS</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>patent</recordtype></control><display><type>patent</type><title>Targeted CRISPR delivery platforms</title><source>esp@cenet</source><creator>ILDAR, Gainetdinov ; IBRAHEIM, Raed ; SONTHEIMER, Erik Joseph ; EDRAKI, Alireza ; XUE, Wen ; MIR, Aamir</creator><creatorcontrib>ILDAR, Gainetdinov ; IBRAHEIM, Raed ; SONTHEIMER, Erik Joseph ; EDRAKI, Alireza ; XUE, Wen ; MIR, Aamir</creatorcontrib><description>The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hvperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide RNA. sequences that permit a complete sgRNA-Nme1Cas9 vector to be inserted into an adeno-associated viral plasmid that is compatible for in vivo administration. Furthermore, Type II-C Cas9 oithologs have been identified that target protospacer adjacent motif sequences limited to between one - four required nucleotides.</description><language>eng</language><subject>BEER ; BIOCHEMISTRY ; CHEMISTRY ; COMPOSITIONS THEREOF ; CULTURE MEDIA ; ENZYMOLOGY ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; SPIRITS ; VINEGAR ; WINE</subject><creationdate>2020</creationdate><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20200611&DB=EPODOC&CC=AU&NR=2018364993A1$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25564,76547</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20200611&DB=EPODOC&CC=AU&NR=2018364993A1$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>ILDAR, Gainetdinov</creatorcontrib><creatorcontrib>IBRAHEIM, Raed</creatorcontrib><creatorcontrib>SONTHEIMER, Erik Joseph</creatorcontrib><creatorcontrib>EDRAKI, Alireza</creatorcontrib><creatorcontrib>XUE, Wen</creatorcontrib><creatorcontrib>MIR, Aamir</creatorcontrib><title>Targeted CRISPR delivery platforms</title><description>The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hvperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide RNA. sequences that permit a complete sgRNA-Nme1Cas9 vector to be inserted into an adeno-associated viral plasmid that is compatible for in vivo administration. Furthermore, Type II-C Cas9 oithologs have been identified that target protospacer adjacent motif sequences limited to between one - four required nucleotides.</description><subject>BEER</subject><subject>BIOCHEMISTRY</subject><subject>CHEMISTRY</subject><subject>COMPOSITIONS THEREOF</subject><subject>CULTURE MEDIA</subject><subject>ENZYMOLOGY</subject><subject>METALLURGY</subject><subject>MICROBIOLOGY</subject><subject>MICROORGANISMS OR ENZYMES</subject><subject>MUTATION OR GENETIC ENGINEERING</subject><subject>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</subject><subject>SPIRITS</subject><subject>VINEGAR</subject><subject>WINE</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2020</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZFAKSSxKTy1JTVFwDvIMDghSSEnNySxLLapUKMhJLEnLL8ot5mFgTUvMKU7lhdLcDMpuriHOHrqpBfnxqcUFicmpeakl8Y6hRgaGFsZmJpaWxo6GxsSpAgAZsiaS</recordid><startdate>20200611</startdate><enddate>20200611</enddate><creator>ILDAR, Gainetdinov</creator><creator>IBRAHEIM, Raed</creator><creator>SONTHEIMER, Erik Joseph</creator><creator>EDRAKI, Alireza</creator><creator>XUE, Wen</creator><creator>MIR, Aamir</creator><scope>EVB</scope></search><sort><creationdate>20200611</creationdate><title>Targeted CRISPR delivery platforms</title><author>ILDAR, Gainetdinov ; IBRAHEIM, Raed ; SONTHEIMER, Erik Joseph ; EDRAKI, Alireza ; XUE, Wen ; MIR, Aamir</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_AU2018364993A13</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng</language><creationdate>2020</creationdate><topic>BEER</topic><topic>BIOCHEMISTRY</topic><topic>CHEMISTRY</topic><topic>COMPOSITIONS THEREOF</topic><topic>CULTURE MEDIA</topic><topic>ENZYMOLOGY</topic><topic>METALLURGY</topic><topic>MICROBIOLOGY</topic><topic>MICROORGANISMS OR ENZYMES</topic><topic>MUTATION OR GENETIC ENGINEERING</topic><topic>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</topic><topic>SPIRITS</topic><topic>VINEGAR</topic><topic>WINE</topic><toplevel>online_resources</toplevel><creatorcontrib>ILDAR, Gainetdinov</creatorcontrib><creatorcontrib>IBRAHEIM, Raed</creatorcontrib><creatorcontrib>SONTHEIMER, Erik Joseph</creatorcontrib><creatorcontrib>EDRAKI, Alireza</creatorcontrib><creatorcontrib>XUE, Wen</creatorcontrib><creatorcontrib>MIR, Aamir</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>ILDAR, Gainetdinov</au><au>IBRAHEIM, Raed</au><au>SONTHEIMER, Erik Joseph</au><au>EDRAKI, Alireza</au><au>XUE, Wen</au><au>MIR, Aamir</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Targeted CRISPR delivery platforms</title><date>2020-06-11</date><risdate>2020</risdate><abstract>The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hvperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide RNA. sequences that permit a complete sgRNA-Nme1Cas9 vector to be inserted into an adeno-associated viral plasmid that is compatible for in vivo administration. Furthermore, Type II-C Cas9 oithologs have been identified that target protospacer adjacent motif sequences limited to between one - four required nucleotides.</abstract><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext_linktorsrc |
| identifier | |
| ispartof | |
| issn | |
| language | eng |
| recordid | cdi_epo_espacenet_AU2018364993A1 |
| source | esp@cenet |
| subjects | BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE |
| title | Targeted CRISPR delivery platforms |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-28T07%3A18%3A46IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-epo_EVB&rft_val_fmt=info:ofi/fmt:kev:mtx:patent&rft.genre=patent&rft.au=ILDAR,%20Gainetdinov&rft.date=2020-06-11&rft_id=info:doi/&rft_dat=%3Cepo_EVB%3EAU2018364993A1%3C/epo_EVB%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_id=info:pmid/&rfr_iscdi=true |