PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD
The present invention relates to the treatment of Pyruvate Kinase Deficiency (PKD) using the Clustered- Regularly Interspaced Short Palindromic Repeats (CRISPR) system. This technology offers the possibility to design an improved single guide RNA (sgRNA), in particular, an improved crRNA to be assoc...
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| creator | FAÑANAS BAQUERO Sara SEGOVIA SANZ José Carlos QUINTANA BUSTAMANTE Óscar PORTEUS Matthew |
| description | The present invention relates to the treatment of Pyruvate Kinase Deficiency (PKD) using the Clustered- Regularly Interspaced Short Palindromic Repeats (CRISPR) system. This technology offers the possibility to design an improved single guide RNA (sgRNA), in particular, an improved crRNA to be associated with tracrRNA, which is incorporated into a CRISPR-associated protein (Cas9) to recognize and induce DNA double-strand breaks at a specific target location. DNA double-strand breaks will be repaired by homologous recombination (HR) in the presence of a donor sequence for PKLR gene repair.
La presente invención se refiere al tratamiento de deficiencia de piruvato quinasa (PKD) utilizando el sistema de repeticiones palindrómicas cortas agrupadas interespaciadas de manera regular (CRISPR). Esta tecnología ofrece la posibilidad de diseñar un ARN guía individual (sgRNA) mejorado, en particular, un crRNA mejorado para ser asociado con tracrRNA, el cual se incorpora en una proteína asociada con CRISPR (Cas9) para reconocer e inducir rompimientos de cadena doble de ADN en una ubicación objetivo específica. Los rompimientos de cadena doble de ADN van a ser reparados mediante recombinación homóloga (HR) en presencia de una secuencia donadora para la reparación del gen de PKLR. |
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La presente invención se refiere al tratamiento de deficiencia de piruvato quinasa (PKD) utilizando el sistema de repeticiones palindrómicas cortas agrupadas interespaciadas de manera regular (CRISPR). Esta tecnología ofrece la posibilidad de diseñar un ARN guía individual (sgRNA) mejorado, en particular, un crRNA mejorado para ser asociado con tracrRNA, el cual se incorpora en una proteína asociada con CRISPR (Cas9) para reconocer e inducir rompimientos de cadena doble de ADN en una ubicación objetivo específica. Los rompimientos de cadena doble de ADN van a ser reparados mediante recombinación homóloga (HR) en presencia de una secuencia donadora para la reparación del gen de PKLR.</description><language>eng ; spa</language><subject>BEER ; BIOCHEMISTRY ; CHEMISTRY ; COMPOSITIONS THEREOF ; CULTURE MEDIA ; ENZYMOLOGY ; HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; SPIRITS ; VINEGAR ; WINE</subject><creationdate>2023</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=20230410&DB=EPODOC&CC=MX&NR=2023000035A$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,776,881,25542,76290</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20230410&DB=EPODOC&CC=MX&NR=2023000035A$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>FAÑANAS BAQUERO Sara</creatorcontrib><creatorcontrib>SEGOVIA SANZ José Carlos</creatorcontrib><creatorcontrib>QUINTANA BUSTAMANTE Óscar</creatorcontrib><creatorcontrib>PORTEUS Matthew</creatorcontrib><title>PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD</title><description>The present invention relates to the treatment of Pyruvate Kinase Deficiency (PKD) using the Clustered- Regularly Interspaced Short Palindromic Repeats (CRISPR) system. This technology offers the possibility to design an improved single guide RNA (sgRNA), in particular, an improved crRNA to be associated with tracrRNA, which is incorporated into a CRISPR-associated protein (Cas9) to recognize and induce DNA double-strand breaks at a specific target location. DNA double-strand breaks will be repaired by homologous recombination (HR) in the presence of a donor sequence for PKLR gene repair.
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| subjects | BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE |
| title | PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD |
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