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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Bibliographische Detailangaben
Hauptverfasser:	PORTEUS, Matthew, FAÑANAS BAQUERO, Sara, SEGOVIA SANZ, José Carlos, QUINTANA BUSTAMANTE, Óscar
Format:	Patent
Sprache:	eng ; fre ; ger
Schlagworte:	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
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