PAH-MODULATING SYSTEMS AND METHODS

The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject. Gene modifying systems for treating phenylketonuria (PKU) are described. L'inv...

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Bibliographische Detailangaben
Hauptverfasser:	STEINBERG, Barrett Ethan, QUERBES, William, NAIR, Rahul Ravindran, COTTA-RAMUSINO, Cecilia Giovanna Silvia, CHIVATAKARN, Onanong, BRODYAGIN, Nikita, APPONI, Luciano Henrique, BRIONES, John Frederick, MIR, Aamir, BOTHMER, Anne Helen, MCALLISTER, Gregory David, WANG, Zhan
Format:	Patent
Sprache:	eng ; fre
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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Beschreibung
Zusammenfassung:	The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject. Gene modifying systems for treating phenylketonuria (PKU) are described. L'invention concerne, par exemple, des compositions, des systèmes et des procédés de ciblage, d'édition, de modification ou de manipulation d'un génome de cellule hôte à un ou plusieurs emplacements dans une séquence d'ADN dans une cellule, un tissu ou chez un sujet. L'invention concerne également des systèmes de modification de gènes pour traiter la phénylcétonurie (PKU).