Researchers in China describe a dual-specific CRISPR-Cas nanosystem for precision therapeutic editing of liver disorders. The system combines liver-targeted delivery and liver-specific editing to precisely treat inflammatory liver diseases. Specific targeting to inflammatory lesions in the liver was achieved by coating nanocomplexes with a layer of biomimetic macrophage membrane. Using the synthetic P3 promoter in plasmids encoding Cas9 or CasRx further ensures liver-specific expression.
Gamida Cell also reported second-quarter financial results and provided a company update. Net loss was $18.6 million, and total cash, cash equivalents and investments amounted to $55.1 million.
Chinese researchers look at the current landscape of gene-editing technology in biomedicine. The review summarises the meaningful applications of CRISPR technologies in preclinical studies, focusing on the establishment of disease models and diagnostic techniques. Moreover, it provides a comprehensive overview of gene-editing attempts for treating various human diseases.
Wednesday, we brought an interview with Verve Therapeutics’ CEO, Sekar Kathiresan. His company recently announced the first human dosing with VERVE-101 - an investigational base-editing therapy designed to treat heterozygous familial hypercholesterolemia. VERVE-101 is the first in vivo base-editing therapy to reach human clinical trials. In the article, Sekar Kathiresan discusses VERVE-101 and how it may transform the lives of individuals with cardiovascular disease.
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