Novel TALE protein scaffolds with improved on/off-target activity ratios

• Main Authors: DUCHATEAU, PHILIPPE, JUILLERAT ALEXANDRE, KAZANCIOGLU, SELINA, BOYEN ALEX
• Format: Patent
• Language: Chinese; English
• Published: 23.07.2024
• 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

The present invention relates to an improved design of TALE protein fusions that can be used as sequence-specific genomic reagents, such as TALE-nuclease and TALE-base editor, exhibiting a higher on-target/out-of-target activity ratio. The object of the invention is the use of producing safer agents for the gene modification of the genome of different types of cells, in particular mammalian cells, in particular for gene therapy. 本发明涉及改进的TALE蛋白融合物的设计，其可用作序列特异性基因组试剂，例如TALE-核酸酶和TALE-碱基编辑器，显示更高的靶上/靶外活性比。本发明的目标是生产更安全的试剂，以对不同类型的细胞特别是哺乳动物细胞的基因组进行基因修饰，特别是用于基因治疗的用途。