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 associ...
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| creator | PORTEUS, Matthew FAÑANAS BAQUERO, Sara SEGOVIA SANZ, José Carlos QUINTANA BUSTAMANTE, Óscar |
| 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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