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Á¦¸ñ [GenScript] Is AAV Based Gene Therapy Safe? New Evidence Says NO!
ÀÛ¼ºÀÚ abbabio
ÀÛ¼ºÀÏÀÚ 2020-01-17
Á¶È¸¼ö 359
 
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Strong evidence from dog studies fueling the safety debate over AAV based gene therapy
Adeno-associated virus (AAV) has been the platform of choice for in vivo gene therapy in recent years. This is not only due to AAV's high efficiency in transducing genes into target tissues, but also its superior safety profile. However, a recent study in dogs with hemophilia showed that AAV can readily insert its payload into host DNA and induce cancer.
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Choose the safer, non-viral, alternative to AAV:
CRISPR RNP + ssDNA HDR Templates
 
Read this case study to learn the advantages of using long single-stranded DNA (ssDNA or ssODN) as non-viral CRISPR HDR templates in cell engineering.
 
¡î  Precise gene knock-in, non-viral, no random insertion
¡î  High gene knock-in efficiency
¡î  Reduced off-target integration
¡î  Low cell toxicity
 
 
GenScript offers high quality, sequence verified long single-stranded DNA (ssDNA or ssODN)  for maximizing the editing efficiency and accuracy of your CRISPR homology directed repair (HDR) based gene knock-in experiments.
151-3000 nt in length;
Sequence verified by sanger sequencing the final ssDNA product;
Up to 20 ug delivery quantity allowing for flexible study design;
Enzymatic approach for ensuring non-detectable levels of dsDNA.
Learn More
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Webinars:
CRISPR Based T Cell Editing: Large knock-ins in human T cells using non-viral HDR templates
Strategies to Efficiently Generate CRISPR KO/KI Cell Lines
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