DISRUPTIVE TECHNOLOGIES
● CRISPR RNAs trigger innate immune responses in human cells
● i-GONAD: a robust method for
in situ
germline genome engineering using CRISPR nucleases
● Long Terminal Repeat CRISPR-CAR coupled ‘universal’ T cells mediate potent anti-leukemic effects
GENOME EDITING AND DISEASES
● Cholesterol halved by CRISPR/Cas9 base editing
● One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system.
● Precise excision of the CAG tract from the Huntingtin gene by Cas9 nickases
● Efficient
in vivo
liver-directed gene editing using CRISPR/CAS9
CLINICAL TRIALS
●
Sangamo announces U.K. authorization of clinical trial evaluating Zinc Finger Nuclease
in vivo
genome editing treatment for Hemophilia B
INDUSTRIAL LANDSCAPE - AGREEMENTS
●
A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent
in vivo
genome editing
● Inscripta raises 55.5MUS$ to advance gene-editing technology