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CRISPR-Cas9 has tremendous potential as a therapeutic tool for treating human diseases. However, prolonged expression of the nuclease and gRNA from viral vectors in an in vivo context may cause off-target activity and immunogenicity. While extracellular vesicles have been recently demonstrated to be...
ORGANISM(S): Homo Sapiens (human) 
CARASIL (cerebral autosomal recessive arteriopathy with subcortical infarcts and leukoencephalopathy) is a hereditary cerebral small-vessel disease caused by loss of function mutations of HTRA1, which is a serine protease with a variety of targets, including extracellular matrix proteins. We isolate...
ORGANISM(S): Mus Musculus (mouse) 
CARASIL (cerebral autosomal recessive arteriopathy with subcortical infarcts and leukoencephalopathy) is a hereditary cerebral small-vessel disease caused by loss of function mutations of HTRA1, which is a serine protease with a variety of targets, including extracellular matrix proteins. Htra1-KO m...
ORGANISM(S): Mus Musculus (mouse) 
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