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in utero delivery of mRNA to the heart,diaphragm and muscle with lipid nanoparticles

Bioactive Materials 2023年第7期25卷 387-398页

作者： Kewa Gao Jie Li Hengyue Song Hesong Han Yongheng Wang Boyan Yin Diana L.Farmer Niren Murthy Aijun Wang Department of Surgery School of MedicineUniversity of CaliforniaDavisSacramentoCA95817United States Department of Bioengineering University of CaliforniaBerkeleyCA94704United States Institute for Pediatric Regenerative Medicine Shriners Hospitals for ChildrenSacramentoCA95817United States Department of Biomedical Engineering University of CaliforniaDavisCA95616United States Department of Burns and Plastic Surgery The Third Xiangya Hospital of Central South UniversityHunan410013China

Nanoparticle-based drug delivery systems have the potential to revolutionize medicine,but their low vascular permeability and rapid clearance by phagocytic cells have limited their medical *** delivered at the in utero stage can overcome these key limitations due to the high rate of angiogenesis and cell division in fetal tissue and the under-developed immune ***,very little is known about nanoparticle drug delivery at the fetal stage of *** this report,using Ai9 CRE reporter mice,we demonstrate that lipid nanoparticle(LNP)mRNA complexes can deliver mRNA in utero,and can access and transfect major organs,such as the heart,the liver,kidneys,lungs and the gastrointestinal tract with remarkable efficiency and low *** addition,at 4 weeks after birth,we demonstrate that 50.99±5.05%,36.62±3.42%and 23.7±3.21%of myofiber in the diaphragm,heart and skeletal muscle,respectively,were ***,we show here that Cas9 mRNA and sgRNA complexed to LNPs were able to edit the fetal organs in *** experiments demonstrate the possibility of non-viral delivery of mRNA to organs outside of the liver in utero,which provides a promising strategy for treating a wide variety of devastating diseases before birth.

关键词： in utero mRNA delivery Nanoparticles Gene editing CRISPR/Cas9

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Efficient delivery of human clotting factor IX after injection of lentiviral vectors in utero

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Acta Pharmacologica Sinica 2004年第6期25卷 87-91页

作者： Xiao-guang CHEN1, Huan-zhang ZHU1, Ju-li GONG, Feng LI, Jing-lun XUE2 State Key Laboratory of Genetic Engineering, Institute of Genetics, Schoolof Life Sciences, Fudan University, Shanghai 200433,China Institute of Genetics School of Life Sciences Fudan University State Key Laboratory of Genetic Engineering 上海 200433

AIM: To explore gene transfer feasibility for human clotting factor IX (hFIX) mediated by recombinant lentivirus in utero. METHODS: ICR mice fetus at 17-19 d gestation were received lentiviral vectors carrying hFIX cDNA under the control of liver specific promoter by intrahepatic injection. The expression and distribution of hFIX cDNA and possible immune responses against the hFIX were assessed by ELISA, PCR, RT-PCR, and immunohistochemistry, respectively. RESULTS: The serum hFIX protein were detected at different time points in all newborn mice, the highest level of hFIX was 50 μg/L and lasted for more than 30 d. Anti-hFIX antibody was not detected. hFIX cDNA was detected in liver, spleen, and heart. The expression of hFIX cDNA was only detected in liver. Besides, no germ line transmission was found at DNA and RNA levels, and no side effect associated with gene transfer was detected. CONCLUSION: The efficient delivery of hFIX can be achieved by prenatal gene transfer. It thus shows the feasibility of gene therapy for hemophilia in utero.

关键词： lentiviral vector factor IX gene transfer techniques in utero

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